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Orphan drugs

4 March 2014

What are orphan drugs?

An orphan drug is defined as a medicine, vaccine or in vivo diagnostic agent that is:

  • intended to treat, prevent or diagnose a rare disease; or
  • not commercially viable to supply to treat, prevent or diagnose another disease or condition.

Medicines need to be designated as orphan drugs by the TGA before an application to register an orphan drug on the Australian Register of Therapeutic Goods (ARTG) will be accepted.

The full definition of an orphan drug can be found in the Therapeutic Goods Regulations 1990, section 16H.

Designated orphan drugs

Before an application to register an orphan drug can be made, a sponsor must first seek orphan drug designation. Information on applications for orphan drug status can be found in the Australian Regulatory Guidelines on Prescription Medicines.

The quality, efficacy and safety of orphan drugs are assessed at the same standard as for other registered medicines.

Drugs designated as orphan drugs by the TGA are shown in the table below. Only those with a date in the 'Date registered in Australia' column can be supplied in Australia.

Note: If you want to print the complete list of orphan drugs, you need to 'Show all' before you print.

A B C D E F G H I J K L M N O P Q R S T U V W X Y Z Show all

A - Orphan drugs

Hint: Click on the table headers to sort the table by a particular column in ascending or descending order.

Drug Sponsor Date of designation Date registered in Australia Indication Dose
Adalimumab (HUMIRA) Abbott Australasia Pty Ltd 16/01/2012 For the treatment of active Crohn's Disease defined as a Paediatric Crohn's Disease Activity Index (PCDAI) score >30 in paediatric patients (6-17 years of age) who have had an inadequate response to conventional therapy, or who are intolerant to or have contraindications for such therapies. Solution for injection
Adalimumab (HUMIRA) AbbVie Pty Ltd 02/11/2012 For the treatment of active Crohn's Disease defined as a Paediatric Crohn's Disease Activity Index (PCDAI) score >30 in paediatric patients (6-17 years of age) who have had an inadequate response to conventional therapy, or who are intolerant to or have contraindications for such therapies. Solution for injection
Adefovir dipivoxil Gilead Sciences Pty Ltd 12/08/2005 In monotherapy or in combination with other therapies, for prevention of recurrence of HBV infection in liver transplant recipients.
Adefovir dipivoxil (HEPSERA) Gilead Sciences Pty Ltd 30/10/2008 For the treatment of chronic hepatitis B in adolescent patients (12 to <18 years of age). 10mg oral tablets
Afamelanotide (SCENESSE) Clinuvel Pharmaceuticals Ltd 08/11/2010 For the treatment of erythropoietic porphyrias. Subcutaneous implant
Afatinib Boehringer Ingelheim Pty Limited 01/06/2012 For the treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with Epidermal Growth Factor Receptor (EGFR) mutation (s). Tablets
Agalsidase alfa (REPLAGAL) PPD Australia Pty Limited 20/10/2000 17/05/2002 For the long-term enzyme replacement therapy in patients with Fabry disease.
Agalsidase beta (FABRAZYME) Genzyme Australasia Pty Ltd 22/05/2000 03/05/2002 For long term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry Disease.
Aimspro (Goat Serum Derived Product) Daval Australia Pty Ltd 22/08/2005 For treatment of Krabbe's Disease (Globoid Cell Leucodystrophy).
Aldesleukin (PROLEUKIN) CSL 16/10/1998 24/05/2000 For the treatment of metastatic renal cell carcinoma.
Aldesleukin (PROLEUKIN) CSL 16/10/1998 To treat metastatic melanoma.
Algucosidase alfa (MYOZYME) Genzyme Australasia Pty Ltd 08/09/2003 For the treatment of Pompe disease (glycogen storage disease type II). Powder for concentrate for solution for infusion
Alpha-1-proteinase inhibitor (GLASSIA) Baxter Healthcare Pty Ltd 03/07/2012 For the treatment of chronic augmentation and maintenance therapy in adults with emphysema due to congenital deficiency of alpha-1-antitrypsin. Solution for IV injection
Ambrisentan GlaxoSmithKline Australia Pty Ltd 05/07/2007 For the treatment of patients with pulmonary arterial hypertension (PAH) to improve exercise capacity, delay clinical worsening and decrease the symptoms of PAH. Tablets 5 mg & 10 mg
5-aminolevulinic acid hydrochloride (Gliolan) Specialised Therapeutics Australia Pty Ltd 30/03/2012 Gliolan is indicated for photodynamic diagnosis of gliomas that are glioblastoma multiforme (GBM) (malignant) on preoperative imaging, and intended for gross macroscopic resection of all visible tumour. Powder for oral solution
Anagrelide hydrochloride (AGRYLIN) Orphan Australia 16/10/1998 23/11/1999 For the treatment of essential thrombocytosis.
Anagrelide hydrochloride (THROMBOREDUCTIN) Orphan Australia Pty Ltd 23/04/2009 For treatment of essential Thrombocythaemia in patients with high or intermediate risk of thrombosis or bleeding. 0.5mg and 1.0mg capsules
Anakinra (KINERET) Invida Asutralia Pty Ltd 20/12/2011 For the treatment of active systemic onset juvenile idiopathic arthritis (SoJIA) in children. Solution for injection
Anakinra (KINERET) Invida Asutralia Pty Ltd 08/05/2013

For the treatment of cryopyrin associated periodic syndromes (CAPS) in adults and children including:

  • Muckle-Wells syndrome (MWS)
  • Familial cold-induced autoinflammatory syndrome (FCAS)/familial cold urticaria (FUC)
  • Neonatal-onset multisystem inflammatory disease (NOMID)/chronic infantile neurological cutaneous and articular syndrome (CINCA)
Solution, pre-filled syringe 100 mg / 0.67 mL
Anti-H5N1 Clinical Network Services Pty Ltd 12/02/2014 Passive immunotherapy in case of post-exposure or suspected post-exposure to H5N1 avian influenza virus. Solution for infusion
Arsenic trioxide Phebra Pty Ltd 24/02/2011 For the treatment of acute promyelocytic leukaemia. Tablets and oral liquid
Arsenic trioxide injection Pharmalab Pty Limited 21/08/2003 For the treatment of patients with acute promyelocytic leukaemia, who are refractory to, or have relapsed from, retinoid and anthracycline chemotherapy.
Arsenic trioxide injection Pharmalab Pty Limited 08/06/2004 For the treatment of patients with acute promyelocytic leukaemia.
Artemether and lumefantrine (RIAMET) Novartis Pharm 27/10/1999 05/07/2002 For the treatment of children and adults with infections due to Plasmodium falciparum or mixed infections including P falciparum.
Artesunate injection Link Pharmaceuticals trading as Link Medical Products Pty Ltd 17/01/2007 For the treatment of severe malaria including cerebral malaria and as a second line treatment in chloroquine resistant malaria cases. Solution for injection
Asfotase alfa Alexion Pharmaceuticals Australasia Pty Ltd 15/10/2012 For the treatment of hypophosphatasia (HPP). Subcutaneous injection
Axitinib Pfizer Australia Pty Ltd 14/04/2011 For the treatment of advanced renal cell carcinoma (RCC). Tablets
5-azacytidine Pharmion Pty Ltd 02/09/2004 For the treatment of myelodysplastic syndrome.
Azacytidine (VIDAZA) Celgene Pty Ltd 17/06/2008 For the treatment of myelodysplastic syndrome. Powder for suspension for injection
Aztreonam lysine Gilead Sciences Pty Ltd 16/07/2007 Control of gram-negative bacteria, particularly pseudomonas aeruginosa, in the respiratory tract of patients with cystic fibrosis. Single-use 1 mL vial containing 75 mg aztreonam and 52.5 mg lysine lyophilized powder, supplied with a single-use diluent ampoule containing 1 mL sterile water for injection with 0.17% NaCl

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B - Orphan drugs

Hint: Click on the table headers to sort the table by a particular column in ascending or descending order.

Drug Sponsor Date of designation Date registered in Australia Indication Dose
Beclometasone dipropionate (ORBEC) Orphan Australia Pty Ltd 31/10/2008 For the treatment of patients with gastrointestinal Graft-Versus-Host-Disease (GVHD) following allogenic haematopoietic cell transplantation in conjunction with an induction course of high-dose prednisone or methylprednisolone. Oral capsules containing 1 mg immediate release beclometasone dipropionate tablet and 1 mg enteric-coated beclometasone dipropionate tablet
Bevacizumab (AVASTIN) Roche Products Pty Ltd 13/01/2009 For the treatment of malignant glioma. A concentrated solution for injection - 100mg in 4 mLs and 400 mg in 16 mLs
BicaVera range of sterile peritoneal dialysis solutions Fresenius Medical Care Australia Pty Ltd 29/10/2002 For use as a peritoneal dialysis solution in the management of end stage renal disease and acute renal failure.
Blinatumomab Amgen Australia Pty Ltd 20/11/2013 For the treatment of B-precursor acute lymphoblastic leukemia. Powder for reconstitution, Injection
Bortezomib (VELCADE) Janssen-Cilag 22/10/2003 08/02/2006 For the treatment of resistant or relapsed multiple myeloma. Powder for injection
Bosentan Omnicare Clinical Research 12/02/2001 12/11/2002 For the treatment of primary pulmonary hypertension or pulmonary hypertension secondary to scleroderma, in patients meeting WHO criteria for inclusion in functional class III-IV pulmonary hypertension.
Bosentan (TRACLEER) Actelion Pharmaceuticals Australia 27/08/2010 For the treatment of digital ulcers in patients with systemic sclerosis. 62.5 mg and 125 mg tablets
Bosutinib Pfizer Australia Pty Ltd 13/08/2012 For the second or third line treatment of chronic myeloid leukaemia. Tablets
Brentuximab vedotin (ADCETRIS) Nycomed Pty Ltd 17/09/2012 & 18/09/2012

For the treatment of:

  • relapsed or refractory Hodgkin lymphoma (HL) and;
  • systemic anaplastic large cell lymphoma (SALCL).
Powder for infusion
Busulfan (BUSULFEX) Orphan Australia Pty Ltd 12/10/2005 13/11/2007 For use in combination with other chemotherapeutic agents and/or radiotherapy as a conditioning regimen prior to haematopoietic progenitor cell transplantation.

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C - Orphan drugs

Hint: Click on the table headers to sort the table by a particular column in ascending or descending order.

Drug Sponsor Date of designation Date registered in Australia Indication Dose
C1 esterase inhibitor (BERINERT P) CSL Limited 10/04/2007 For the treatment of hereditary angioedema. Powder for injection
C1 esterase inhibitor (CINRYZE) Cedarglen Investments Pty Ltd 15/10/2010 For the treatment, routine prevention and pre-procedure prevention of angiodema attacks in adults, adolescents, and children from 6 years of age with C1 inhibitor deficiency. Powder for reconstitution for intravenous administration
Caffeine citrate injection Ophtalmic Laboratories Ltd 02/08/2007 For the short term treatment of apnoea of prematurity of gestational age 28 to less than 33 weeks. Solution for injection 40 mg/2mL
Caffeine citrate oral solution Ophtalmic Laboratories Ltd 02/08/2007 For the short term treatment of apnoea of prematurity of gestational age 28 to less than 33 weeks. 25mg/5mL
Canakinumab (ILARIS) Novartis Pharmaceuticals Australia Pty Limited 01/05/2013 For the treatment of active Systemic Juvenile Idiopathic Arthritis (sJIA) in patients aged 2 years and older. Powder for Injection
Caprine hyperimmune serum against HIV lysate (AIMSPRO) Daval Australia Pty Ltd 15/05/2009 For the treatment of amyotrophic lateral sclerosis. 4.5mg total protein/ml liquid for subcutaneous injection
Carboprost as the tropmethamine (HEMABATE) Pharmacia & Upjohn 16/10/1998 07/06/2001 For the treatment of post-partum haemorrhage due to uterine atony.
Carglumic acid (CARBAGLU™) Emerge Health Pty Ltd 13/03/2012

For the treatment of the following:

  • hyperammonaemia due to N-acetylglutamate synthase primary deficiency;
  • hyperammonaemia due to isovaleric acidaemia;
  • hyperammonaemia due to methylmalonic acidaemia and;
  • hyperammonaemia due to propionic acidaemia.
Dispersible tablet
Carmustine (GLIADEL) Orphan Australia Pty Ltd 25/09/2003 27/10/2004 For use as an adjunct to surgery to prolong survival in patients with malignant glioma.
Carmustine implants (GLIADEL wafers) Rhone-Poulenc Rorer Australia 16/10/1998 29/12/2000 For use as an adjunct to surgery to prolong survival in patients with recurrent glioblastoma multiforme for whom surgical resection is indicated
Catridecacog Novo Nordisk Pharmaceuticals Pty Ltd 22/11/2011 Routine prophylaxis for bleeding in patients with congenital Factor XIII A subunit deficiency. Powder for injection
Catumaxomab (REMOVAB) Biotech Regulatory Solutions 15/09/2010 For the treatment of patients with malignant ascites due to EpCAM-positive carcinomas Concentrate for solution for infusion
Celecoxib (CELEBREX) Searle 10/04/2000 17/08/1999 To reduce the number of adenomatous colorectal polyps in familial adenomatous polyposis (FAP), as an adjunct to usual care (eg endoscopic surveillance, surgery).
6-chloro-2, 3, 4, 9-tetrahydro-1H-carboxamide Siena Biotech Italy 11/03/2011 For the treatment of Huntington's Disease.
Cholic acid Orpharma Pty Ltd 25/07/2013 For the treatment of inborn errors of bile acid synthesis responsive to treatment with cholic acid. oral capsules 50 mg and 250 mg
Cladribine (LITAK) Orphan Australia Pty Lt 23/01/2003 22/06/2004 For the treatment of hairy cell leukaemia and lymphoplasmacytic lymphoma (Waldenstrom's macroglobulinaemia).
Clobazam (FRISIUM) Sanofi-Aventis Australia Pty Ltd 14/09/2009 For the treatment of paediatric refractory epilepsy. Tablet
Clofarabine (EVOLTRA) Hospira Australia Pty Ltd 02/10/2007 For the treatment of acute myeloid leukaemia in adults 60 years of age and over and for the treatment of acute lymphoblastic leukaemia in paediatric patients who have relapsed or are refractory after receiving at least two prior regimens. Concentrated solution for infusion
Colistimethate sodium Phebra Pty Ltd 31/10/2008 For treatment of pseudomonas aeruginosa infections in patients with cystic fibrosis. Sterile lyophilised powder 1MIU and 2MIU (colistimethate base) in single use vials for nebulisation.
Combivir GlaxoSmithKline Australia 10/07/2007 For antiretroviral combination therapy for the treatment of Human Immunodeficiency Virus (HIV) infected children under 12 years of age. Tablets (containing lamivudine 150 mg & zidovudine 300 mg)
Cyclosporin Delpharm Consultants Pty Ltd 17/11/2004 For the prevention of graft rejection of lung transplants.
Cytarabine intrathecal injection (DEPOCYT) Orphan Australia Pty Ltd 07/01/2004 28/06/2006 For the treatment of symptomatic neoplastic meningitis. 50mg/5mL injection vial

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D - Orphan drugs

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Drug Sponsor Date of designation Date registered in Australia Indication Dose
Dabrafenib GlaxoSmithKline 30/05/2012 For the treatment of patients with BRAF V600 mutation positive unresectable or metastatic (Stage IV) melanoma. Capsules
Darunavir (PREZISTA) Janssen-Cilag Pty Ltd 22/08/2008 For the treatment of HIV infection in paediatric patients (children and adolescents aged 6 to <18 years). 75 mg, 150 mg, 300 mg and 600 mg tablet
Dasatinib (SPRYCEL) Bristol-Myers Squibb Pharmaceuticals 12/10/2005 &
18/01/2006
09/01/2007
  • For the treatment of chronic myeloid leukaemia in patients who are resistant to or intolerant of imatinib.
  • For Philadelphia-positive acute lymphoblastic leukaemia (Ph+ ALL).
Tablets
Decitabine (DACOGEN) Janssen-Cilag Pty Ltd 16/10/2012 For the treatment of adult patients ≥65 years with newly diagnosed de novo or secondary acute myeloid leukaemia (AML). Injection
Deferasirox (EXJADE) Novartis Pharmaceuticals Australia Pty Ltd 24/09/2004 30/06/2006 For the treatment of chronic iron overload in patients with transfusion-dependent anaemias. Dispersible tablets
Deferiprone (FERRIPROX) Orphan Australia Pty Ltd 22/05/2001 02/04/2003 For the treatment of iron overload in patients with thalassaemia major, unwilling or unable to take desferrioxamine therapy.
Defibrotide Link Healthcare 12/11/2013 For the treatment of hepatic veno-occlusive disease (VOD). Concentrate for solution for infusion
Denosumab (XGEVA) Amgen Australia Pty Ltd 03/10/2012 For the treatment of patients with giant cell tumours of the bone. Injection
DEXPRAMIPEXOLE Biogen Idec Australia Pty Limited 25/10/2011 For the treatment of amyotrophic lateral sclerosis (ALS). Tablets
Dornase alpha (PULMOZYME) Roche Products Pty Limited 28/04/2004 For the management of demonstrated respiratory complications in cystic fibrosis in children under the age of five years.
Drisapersen GlaxoSmithKline 10/12/2013 For the treatment of patients with Duchenne muscular dystrophy (DMD) bearing certain mutations that are amenable to treatment with exon 51 skipping. Solution for injection
Duodopa (LEVODOPA / CARBIDOPA) Abbott Products Pty Ltd 16/12/2010 For the treatment of advanced idiopathic Parkinson's disease with severe motor fluctuations and not responding to oral treatment. 20mg/mL and 5mg/mL intestinal gel

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E - Orphan drugs

Hint: Click on the table headers to sort the table by a particular column in ascending or descending order.

Drug Sponsor Date of designation Date registered in Australia Indication Dose
Ecallantide (KALBITOR) Link Medical Products Pty Ltd 13/05/2011 For the treatment of patients with hereditary angioedema. Solution for Injection
Eculizumab (rmc) (SOLIRIS) Alexion Pharmaceuticals Australasia Pty Ltd 05/09/2011 For the treatment of atypical haemolytic uraemic syndrome (aHUS). Solution for intravenous infusion 300mg in 30 mL vial
Eculizumab (SOLIRIS) Alexion Pharmaceuticals Australasia Pty Ltd 26/10/2012 For the treatment of infection - associated haemolytic uraemic syndrome. Concentrated solution for IV infusion vial
Eculizumab (SOLIRIS) Cedarglen Investments Pty Limited 10/04/2007 For the treatment of paroxysmal nocturnal haemoglobinuria. solution for injection E
Efavirenz (STOCRIN) Merck Sharp & Dohme 31/10/2000 16/08/2002 For the treatment of HIV-1 infection in children and adolescents over the age of 3 years.
ELAPRASE idursulfase-rhu Solution For IV Infusion vial Genzyme Australasia Pty Ltd 30/06/2006 21/01/2008 For the long term treatment of patients with Hunter syndrome (Mucopolysaccharidosis II, MPS II).
Eliglustat Sanofi-Aventis Australia Pty Ltd 02/08/2013 The indication is for the treatment of long term treatment of adult patients with Gaucher disease type 1 (GD1) Capsules
Elosulfase-Alpha  BioMarin Pharmaceutical Australia Pty Ltd  05/07/2013    For the treatment of mucopolysaccharidosis IV type A (Morquio A syndrome, MPS IVA).  Solution for infusion 
Eltrombopag olamine (REVOLADE) GlaxoSmithKline Australia Pty Ltd 17/07/2008 For the treatment of idiopathic thrombocytopenic purpura (ITP). Tablet
Epoprostenol (VELETRI) Actelion Pharmaceuticals Australia Pty Ltd 17/01/2012 Treatment of pulmonary arterial hypertension. Lyophilized powder for reconstitution
Epoprostenol sodium (FLOLAN) Glaxo Wellcome 06/12/1999 19/09/2001 For the long term treatment of primary pulmonary hypertension in New York Heart Association (NYHA) Class III and IV patients.
Epoprostenol sodium (FLOLAN) GlaxoSmithKline Pty Ltd 05/04/2006 For the treatment of Pulmonary Arterial Hypertension (PAH) associated with collagen vascular diseases, in New York Heart Association (NYHA) functional class III and class IV patients.
Eptacog alfa (NOVOSEVEN) Novo Nordisk Pharmaceuticals Pty Ltd 14/01/2005 For the prevention and treatment of bleeding episodes in patients with congenital Factor VII deficiency or Glanzmann's Thrombasthenia.
Erlontinib (TARCEVA) Roche Products Pty Ltd 14/04/2011 For the treatment of advanced (Stage IIIB) or metastatic (Stage IV) non-small cell lung cancer (NSCLC) with activating EGFR mutations. Tablets
Ethyl eicosapentaenoate Orphan Australia Pty Ltd 15/07/2004 For the treatment of Huntington's Disease.
Everolimus (AFINITOR) Novartis Pharmaceuticals Australia Pty Ltd 17/07/2008 For the treatment of advanced renal cell carcinoma (RCC). 5 mg and 10 mg tablets
Everolimus (AFINITOR) Novartis Pharmaceuticals Australia Pty Ltd 26/07/2010 For the treatment of tuberous sclerosis complex (TSC), including SEGA, angiomyolipoma and lymphangioleiomyomatosis (LAM) associated with TSC. 5 mg and 10 mg tablets
Extraneal brand of icodextrin 7.5% w/v peritoneal dialysis solution Baxter Healthcare Pty Ltd 26/09/2001 17/10/2002 For use as an alternative osmotic agent in dialysis solutions for long dwell exchange in patients treated with peritoneal dialysis.
Ex vivo cultured adult human mesenchymal stem cells (PROCHYMAL) Delpharm Consultants Pty Ltd 08/11/2010 For the treatment of acute Graft versus Host Disease (aGvHD) in patients who have undergone Haematopoietic Stem Cell Transplantation (HSCT). Solution for intravenous infusion

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F - Orphan drugs

Hint: Click on the table headers to sort the table by a particular column in ascending or descending order.

Drug Sponsor Date of designation Date registered in Australia Indication Dose
Factor eight inhibitor bypassing activity (FEIBA VH) Baxter Healthcare Pty Limited 20/05/2004 For the treatment of haemophilia A patients with Factor VIII inhibitor and the treatment of hameophilia B patients with Factor IX inhibitor.
Factor VIII[rDNA]; (KOGENATE FS, SF; HELIXATE FS, SF) Bayer 17/06/1999 28/02/2001 For the treatment and prophylaxis of bleeding in haemophilia A (congenital factor VIII deficiency) in previously treated and untreated patients, and in patients with Factor VIII inhibitors (neutralising antibodies) who continue to respond to Kogenate FS (ie in whom haemostasis is achieved) [Kogenate FS does not contain von Willebrand's Factor and hence is not indicated in von Willebrand's disease].
Fedratinib Sanofi-Aventis Australia Pty Ltd For the treatment of primary myelofibrosis, of post-polycythaemia vera myelofibrosis and of post-essential thrombocythaemia myelofibrosis. Capsules
FEIBA NF factor eight inhibitor bypassing fraction 500U powder For injection vial with diluent vial Baxter Healthcare Pty Ltd 22/06/2004 13/12/2006 Is indicated as second line therapy for the control of spontaneous bleeding episodes and use in surgery in haemophilia A or B patients with inhibitors, for whom recombinant human factor VIIa has failed, is contraindicated or has been associated with unacceptable adverse effects.
Fluocinolone acetonide intravitreal implant (RETISERT) Bausch & Lomb 12/12/2005 For treatment of chronic, non-infectious uveitis affecting the posterior segment of the eye.
Fomivirsen sodium (VITRAVENE) Ciba Vision Australia Pty Ltd 01/04/1999 02/05/2000 For the local treatment of cytomegalovirus (CMV) retinitis in patients with acquired immunodeficiency (AIDS).
Forodesine (FOSODINE) Mundipharma Pty Ltd 24/03/2009 For the treatment of cutaneous T-cell lymphoma (CTCL). 100mg tablets and 5 mg/mL solution for injection

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G - Orphan drugs

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Drug Sponsor Date of designation Date registered in Australia Indication Dose
G17DT (IMMUNOGEN) Kendle R & D Pty Ltd 20/11/2003 For the treatment of gastric and pancreatic cancers.
Galsulfase rch (NAGLAZYME)
Cedarglen Investments Pty Ltd 18/01/2006 16/02/2007 For long-term enzyme replacement therapy in the treatment of patients with mucopolysaccharidosis type VI (MPS VI). Solution for injection
Gemtuzumab ozogamicin (MYLOTARG) Wyeth Australia Pty Ltd 25/10/2000 For the treatment of acute myeloid leukaemia.
GENOTROPIN brand of somatropin (rbe) Pfizer Australia Pty Ltd 09/02/2007 For the treatment of growth disturbance in children with chronic renal insufficiency. Powder for injection

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H - Orphan drugs

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Drug Sponsor Date of designation Date registered in Australia Indication Dose
Histamine dihydrochloride FH Faulding and Co Limited 25/10/2000 For the treatment of metatastic melanoma (stage IV).
Human fibrinogen (RIASTAP) CSL Limited 15/05/2009 For the treatment of congenital fibrinogen deficiency. Powder for injection by intravenous infusion
Human hepatitis B immunoglobulin (HEPATECT CP) Bioregulatory Consulting 03/06/2010 Prophylaxis against re-infection of a transplanted liver in patients who carry the surface antigen of the hepatitis B virus. Solution for intravenous infusion
Human hepatitis B immunoglobulin (HEPATECT CP) Link Medical Products Pty Ltd 27/08/2009 Prophylaxis against re-infection of a transplanted liver in patients who carry the surface antigen of hepatitis B virus.
Human plasma-derived protein C (CEPROTIN) Baxter Healthcare Pty Limited 23/01/2003 For the treatment of severe congenital protein C deficiency.
Humatrope brands of somatropin powder for subcutaneous or intramuscular injection Eli Lilly Australia Pty Ltd 13/06/2002 For the treatment of growth failure in children born small for gestational age.

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I - Orphan drugs

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Drug Sponsor Date of designation Date registered in Australia Indication Dose
Ibuprofen (PEDEA) Emerge Heath Pty Ltd 29/05/2013 For the treatment of haemodynamically significant patent ductus arteriosus in preterm newborn infants less than 34 weeks of gestational age. Solution for Injection
Icatibant (FIRAZYR) Shire Australia Pty Ltd 10/10/2013 Treatment of angiotensin-converting-enzyme inhibitor (ACE-1) induced angioedema. Solution for injection
Icatibant (FIRAZYR) Shire Australia Pty Ltd 12/02/2009 For the treatment of acute attacks of hereditary angioedema. 30mg/3mL pre-filled syringe
Icodextrin 7.5% (EXTRANEAL PERITONEAL DIALYSIS SOLUTION) Baxter Healthcare Pty Ltd 16/10/1998 16/12/1999 For the treatment of life threatening, End Stage Renal Disease, for those patients who have been identified as requiring the specialised treatment of peritoneal dialysis, and who have subsequently demonstrated significantly reduced ultrafiltration with intraperitoneal hyperosmolar glucose.
Iloprost trometamol (ILOMEDIN) Schering P/L 13/10/2000 For the treatment of primary pulmonary hypertension.
Imatinib mesylate (GLIVEC) Novartis Australia Pty Ltd 12/05/2011 For the treatment of pulmonary arterial hypertension. Tablets and capsules
Imatinib mesylate (GLIVEC) Novartis Pharmaceuticals Australia Pty Ltd 12/08/2002 28/02/2003 For the treatment of chronic myeloid leukaemia.
Imatinib mesylate (GLIVEC) Novartis Pharmaceuticals Australia Pty Ltd 10/12/2001 21/06/2002 For the treatment of adult patients with unresectable and/or metastatic malignant gastrointestinal stromal tumours.
Imatinib mesylate (GLIVEC) Novartis Pharmaceuticals Australia Pty Ltd 01/10/2008 For use as adjuvant therapy For gastrointestinal stromal tumour (GIST). 5 mg & 100 mg capsules and 100 mg & 400 mg tablets
Imatinib mesylate (GLIVEC) Novartis Pharmaceuticals Australia Pty Ltd 03/05/2006

For the treatment of:

  • Philadelphia-positive acute lymphoblastic leukaemia (Ph+ ALL)
  • Dermatofibrosarcoma protuberans (DFSP)
  • Myelodysplastic/myeloproliferative diseases (MDS/MPD) associated with platelet-derived growth factor receptor (PDGFR) gene rearrangements
  • Idiopathic hypereosinophilic syndrome including acute and chronic eosinophilic leukaemia
  • Systemic mastocytosis without the D816V c-kit mutation.
Imiglucerase (CEREZYME) Genzyme Australia 16/10/1998 17/05/1999 For enzyme replacement for patients with a confirmed diagnosis of Gaucher's disease.
Immunoglobulin - antithymocyte (rabbit) (ATG-FRESENIUS) Dutec Diagnostics Pty Ltd 18/06/2010 For the prevention of graft versus host disease in allogeneic haematopoietic stem cell transplantation. Solution for intravenous injection
Indinavir sulfate (CRIXIVAN) Merck Sharp & Dohme 03/10/2000 18/09/2001 For the treatment of HIV-1 infection in children and adolescents over the age of 3 years.
Infliximab Schering-Plough 12/10/2005 For reducing the signs and symptoms in patients with moderately to severely active polyarticular-course juvenile rheumatoid arthritis (JRA) who have had an inadequate response to methotrexate.
Ivacaftor (KALYDECO) Vertex Pharmaceuticals 14/03/2012 For the treatment of cystic fibrosis (CF) in patients who have a gating or an R117H mutation in the CFTR gene. Tablets -film coated 150mg

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J - Orphan drugs

There are no orphan drugs beginning with 'j'.

K - Orphan drugs

There are no orphan drugs beginning with 'k'.

L - Orphan drugs

Hint: Click on the table headers to sort the table by a particular column in ascending or descending order.

Drug Sponsor Date of designation Date registered in Australia Indication Dose
Lamivudine (ZEFFIX) Glaxosmithkline 19/06/2002 For the treatment of children (2 years and above) and adolescents with chronic hepatitis B and evidence of hepatitis B virus (HBV) replication. This indication is based on changes in serological and histological markers in clinical studies up to 18 months duration in patients with compensated liver disease.
Lamivudine (ZEFFIX 3TCTM)
Glaxo Wellcome Australia Ltd 11/01/2001 18/12/2001 For use in combination with other antiretroviral agents for the treatment of HIV infection in children.
Lanreotide (SOMATULINE AUTOGEL) Ipsen Pty Limited 19/03/2004 17/12/2004 For the treatment of the symptoms of carcinoid syndrome associated with carcinoid tumours and the symptoms of VIPomas.
Laronidase (ALDURAZYME) Genzyme Australiasia Pty Ltd 08/08/2001 04/05/2004 For the treatment of mucopolysaccharidosis.
Leflunomide Aventis Pharma 08/04/2004 For treatment of polyarticular course juvenile rheumatoid arthritis in patient 3 to 17 years of age.
Lenalidomide (REVLIMID) Celgene Pty Ltd 06/09/2006 13/12/2007

For the treatment of :

  • previously treated multiple myeloma patients in combination with dexamethasone; and
  • patients with transfusion-dependent anaemia due to low or intermediate-1 risk myelodysplastic syndromes associated with a deletion 5q cytogenetic abnormality, with or without additional cytogenetic abnormalities.
Capsules
Lepirudin (REFLUDAN) Celgene Pty Ltd 09/10/2008 For the treatment of acute heparin associated thrombocytopenia (HAT) type II patients with thrombocytopenia or thromboembolic complications and for the prophylaxis of thrombocytopenia or new thromboembolic events in patients with a history of HAT type II and a need for effective antithrombotic measures (e.g. during open heart surgery). 50mg powder for injection
Lepirudin (REFLUDAN) Hoechst Marion Roussel 16/10/1998 For the treatment of acute heparin associated thrombocytopenia (HAT) type II patients with thrombocytopenia or thromboembolic complications and for the prophylaxis of thrombocytopenia or new thromboembolic events in patients with a history of HAT type II and a need for effective antithrombotic measures (e.g. during open heart surgery).
Leuprorelin Acetate (LUCRIN) AbbVie Pty Ltd 19/12/2012 For the treatment of Central Precocious Puberty (CPP). Injection
Linezolid (ZYVOX) Pharmacia Australia Pty Ltd 09/01/2003 For the treatment of children, from birth to less than 18 years, with suspected or proven infections due to organisms resistant to multiple classes of antibiotics, including methicillin resistant Staphylococcus species and vancomycin resistant Enterococcus species.

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M - Orphan drugs

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Drug Sponsor Date of designation Date registered in Australia Indication Dose
Macitentan Actelion Pharmaceuticals Australia Pty Limited 13/07/2012 For the treatment of pulmonary arterial hypertension. Film coated tablets
Mannitol (BRONCHITOL) Pharmaxis Ltd 16/04/2009 For the treatment of patients with cystic fibrosis to improve lung function and reduce pulmonary exacerbations. 40mg powder for inhalation
Mecasermin [rhIGF-1] injection (INCRELEX) 10mg/ml in 40 mg vial Ipsen Pty Ltd 01/07/2008 For the long-term treatment of growth failure in children and adolescents with severe primary insulin-like growth factor-1 deficiency (severe primary IGFD). Solution for subcutaneous injection
Melphalan (ALKERAN INJECTION) Glaxo Wellcome 22/03/1999 04/09/2001 For the treatment of multiple myeloma in patients For whom oral therapy is inappropriate.
Mepolizumab (BOSATRIA) GlaxoSmithKline Australia Pty Ltd 17/03/2008 For the treatment of patients with hypereosinophilic syndrome Powder for injection
Mercaptopurine Ballia Holdings Pty Ltd 14/12/2012 For the treatment of Acute Lymphoblastic Leukaemia in Children. Oral suspension
Mercaptopurine (XALUPRINE) Link Medical Products Pty Ltd 15/08/2012 For the treatment of Acute Lymphoblastic Leukaemia in paediatric patients. Oral suspension
Methotrexate Ballia Holdings Pty Ltd 14/12/2012 For the treatment of Acute Lymphoblastic Leukaemia in Children. Oral suspension
Midazolam (MIDAZOLAM BUCCAL LIQUID) Phebra Pty Ltd 01/06/2009 For the treatment of status epilepticus in children and adolescents aged 0 to 19 years. 10mg in 1mL liquid to be administered via an oral syringe into the buccal cavity
Mifamurtide (Mepact) Nycomed Pty Ltd 02/09/2013 Powder for infusion
Miglustat (ZAVESCA) Actelion Pharmaceuticals Australia Pty Ltd 11/07/2005 02/03/2007 For the treatment of mild to moderate type 1 Gaucher disease. Capsules
Miglustat (ZAVESCA) Actelion Pharmaceuticals Australia Pty Ltd 08/07/2008 For the treatment of Niemann-Pick disease Type C. Capsules
M-iodobenzyl [123I] guanidine injection (MIBGEN) Australian Nuclear Science and Technology Organisation (ANSTO) 15/08/2002 26/05/2004

For:

  • Diagnostic scintigraphic localisation of tumours originating in tissue that embryologically stems from the neural crest. These are neuroblastomas, pheochromocytomas, paragangliomas, chemodectomas and ganglioneuromas.
  • Detection, staging and follow-up on therapy of neuroblastomas.
  • Diagnosis of neural crest tumours, including those arising from the adrenal medulla, by evaluation of the uptake of iobenguane.
Mometasone furoate RhinoScience Pty Ltd 14/09/2011 For the treatment of persistent post-surgical eosinophilic polypoid chronic rhinosinusitis. Irrigation base equivalent 2mg/240mL solution
MultiBic Fresenius Medical Care Australia Pty Ltd 16/08/2004 For the treatment of acute renal failure in intensive care units.
Mycophenolic acid (as sodium) MYFORTIC Novartis Pharmaceuticals Pty Ltd 11/01/2010 For induction and maintenance treatment of lupus nephritis. 180 mg and 360 mg enteric coated tablets

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N - Orphan drugs

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Drug Sponsor Date of designation Date registered in Australia Indication Dose
Nandrolone decanoate (DECA-DURABOLIN 100) Organon 30/12/1999 For patients who have proven HIV infection who have lost at least 5% of their usual body weight.
Nanoparticle albumin-bound paclitaxel (ABRAXANE) Abraxis BioScience Australia Pty Ltd 01/12/2009 For treatment of pancreatic cancer. Powder for injection (suspension)
Nevirapine (VIRAMUNE) Boehringer Ingelheim Pty Limited 11/03/2003 For the prevention of mother to child transmission of HIV-1 in pregnant women who are not taking antiretroviral therapy at time of labour.
Nilotinib (TASIGNA) Novartis Pharmaceuticals Australia Pty Ltd 09/06/2006 14/01/2008 For treatment of patients with chronic myeloid leukaemia. 200mg capsules blister packs
Nitazoxanide (CRYPTAZ) Tri-Med Distributors 13/10/1999 For the treatment of patients with AIDS who are positive for a stool test for cryptosporidiosis.
Nitisinone (ORFADIN) Orphan Australia Pty Ltd 30/10/2008 For the treatment of hereditary tyrosinaemia type 1. 2mg, 5mg and 10mg capsules
Nitric Oxide (INOmax) Ikaria Australia Pty Ltd 11/09/2013 The indication is that it is indicated, in conjunction with ventilator support and other appropriate agents, as part of the treatment of peri-and post-operative pulmonary hypertension in newborn infants, infants and toddlers, children and adolescents, ages 0-17 years in conjunction with heart surgery, in order to selectively decrease pulmonary arterial pressure and improve right ventricular function and oxygenation. indication is inhalational gas
Nitric oxide (INOmax) Delpharm Consultants Pty Limited 11/05/2006 16/11/2007 For the treatment of Persistent Pulmonary Hypertension (PPH) in neonates. 800 ppm medicinal gas for inhalation cylinder
Normal immunoglobulin (human) (KIOVIG) Baxter Healthcare Pty Ltd 29/11/2012 For the treatment of immunomodulatory therapy in multifocal motor neuropathy (MMN). Intravenous use injection
Normal immunoglobulin (INTRAGLOBIN F) Medical Dynamics Australia (MDA) Pharma 27/02/2001 11/04/2001

For the treatment of:

  • Primary Immune Deficiency Syndromes
  • Kawasaki disease
  • Paediatric HIV infection / Congenital AIDS
  • Allogeneic Bone Marrow Transplantation
  • Idiopathic Thrombocytopaenic Purpura.
NOVOSEVEN brand of eptacog alfa (activated) bhk Novo Nordisk Pharmaceuticals Pty Ltd 30/01/2007 For the treatment of postpartum haemorrhage in patients unresponsive to standard obstetrical management, oxytocic drugs and standard blood component therapy prior to major invasive therapy. Powder for injection
NSC-631570 (UKRAIN) Mrs Aleksandra Harasemcuk 08/06/2004 For the treatment of pancreatic cancer.

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O - Orphan drugs

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Drug Sponsor Date of designation Date registered in Australia Indication Dose
Oblimersen (GENASENSE) ICON Clinical Research 10/01/2008 For the treatment of patients with stage IV malignant melanoma. Solution for infusion
Oblimersen (GENASENSE) Kendle R and D Pty Ltd 24/11/2006 For the treatment of patients with stage IV malignant melanoma.
Oblimersen (GENASENSE) Aventis Pharma Pty Ltd 25/09/2003 For the treatment of patients with stage IV malignant melanoma.
Octocog alfa rch (ADVATE) intravenous injection Baxter Healthcare Pty Ltd 19/02/2007 05/12/2007 Is indicated for use in haemophilia A for prevention and control of haemorrhagic episodes.
Octocog alfa rch (ADVATE) powder for injection vials with diluent Baxter Healthcare Pty Ltd 10/03/2004 25/02/2005 Is indicated for use in haemophilia A for prevention and control of haemorrhagic episodes.
Octreotide (SANDOSTATIN LAR) Novartis Pharmaceuticals Australia Pty Ltd 15/09/2010 For the treatment of patients with advanced neuroendocrine tumours of the midgut or unknown primary tumour location. 10, 20 and 30 mg prefilled syringe composite packs
Octreotide (SANDOSTATIN / SANDOSTATIN LAR) Novartis Pharmaceuticals Australia Pty Ltd 15/05/2009

For treatment of patients with symptoms associated with:

  • functional gastro-entero-pancreatic neuroendocrine tumours (GEP/NETs);
  • Glucagonomas;
  • Gastrinomas/Zollinger-Ellison syndrome;
  • Insulinomas, for pre-operative control of hypoglycaemia and for maintenance therapy; and
  • GRFomas.
  • 500 microgram/1mL injection ampoule
  • 50 microgram/1mL injection ampoule
  • 100 microgram/1mL injection ampoule
  • 10mg injection vial plus diluent prefilled syringe composite pack
  • 20mg injection vial plus diluent prefilled syringe composite pack and;
  • 30mg injection vial plus diluent prefilled syringe composite pack
Olaparib AstraZeneca Pty Ltd 15/01/2014 Is indicated as monotherapy for the maintenance treatment of adult patients with platinum-sensitive relapse BRCA-mutated ovarian, fallopian tube or primary peritoneal cancer who are in response to platinum-based chemotherapy. Capsule
Oprelvekin (IL-11) (NEUMEGA) Wyeth 07/04/1999 For the prevention of severe chemotherapy-induced thrombocytopenia (SCIT) in patients with non-myeloid malignancies who are judged by their physician to be at risk of severe thrombocytopenia.
Ovine anti-colchicine polyclonal antibody fragments (COLCHIFAB) Microfarm Limited 04/07/2011 As an antidote in cases of acute colchinine toxicity. Ampoules

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P - Orphan drugs

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Drug Sponsor Date of designation Date registered in Australia Indication Dose
Panobinostat (TBA) Novartis Pharmaceuticals Australia Pty Ltd 24/03/2009 For the treatment of cutaneous T-cell lymphoma (CTCL). Capsules
Pasireotide (SIGNIFOR LAR) Novartis Pharmaceuticals Australia Pty Ltd 29/10/2013 For the treatment of patients with Acromegaly for whom medical therapy is appropriate. Modified release injection
Pasireotide (SIGNIFor) Novartis Pharmaceuticals Australia Pty Limited 03/08/2011 For the treatment of patients with Cushing's disease for whom medical therapy is appropriate. Solution for injection
Pazopanib (VOTRIENT) GlaxoSmithKline Australia Pty Ltd 13/05/2011 For the treatment of advanced (unresectable and/or metastatic) soft tissue sarcoma. Tablets
Pazopanib (PATORMA) GlaxoSmithKline Australia Pty Ltd 24/03/2009 For the treatment of advanced and/or metastatic renal cell carcinoma (RCC). 200mg and 400mg film coated tablets
PEDITRACE and ADDAMEL N brands of trace element solutions for intravenous injection Fresenius Medical Care Australia Pty Ltd 08/11/2001 For the supply of basal trace element requirements in infants/children and adults, respectively.
Pegaspargase (PEGASPAR) Rhone-Poulenc Rorer 20/01/1999 For the treatment of patients with acute lymphoblastic leukaemia who require L-asparaginase in their treatment regimen, but have developed hypersensitivity to the native forms of L-asparaginase.
Peginterferon alfa-2a and ribavirin (PEGASYS RBV) Roche Products Pty Limited 15/06/2012 For the treatment of chronic hepatitis C in treatment-naïve children and adolescents 5 years of age and older with compensated liver disease. Injection
Peginterferon alfa-2b and ribavirin (PEGATRON COMBINATION THERAPY) Schering-Plough Pty Limited 15/01/2010 For the treatment of chronic hepatitis C in children = 27 kg with compensated liver disease and who have not received previous interferon treatment. Peginterferon alfa-2b; 50, 80, 100, 120 and 150 microgram powder for injection and ribavirin 200 mg capsule.
Pegvisomant (rbe) (SOMAVERT) Pharmacia Australia Pty Ltd. 09/10/2003 07/12/2005 For the treatment of patients with acromegaly. Powder for injection with diluent
Pegylated liposomal doxorubicin hydrochloride (CAELYX) Schering-Plough 28/02/2000 21/09/2001 For the treatment of advanced ovarian cancer in women who have failed first-line platinum-based chemotherapy.
Pegylated liposomal doxorubicin (CAELYX) Schering-Plough Pty Ltd 05/07/2007 For use, in combination, for the treatment of progressive multiple myeloma in patients who have received at least one prior therapy and who have undergone or are unsuitable for bone marrow transplantation. Injection vial 20 ml/10mL and 50mg/25mL
Pentostatin (NIPENT) Hospira Australia Pty Ltd 15/05/2009 For the treatment of Hairy Cell Leukaemia (HCL) 10mg powder for injection and powder for solution for infusion
Peritoneal dialysis solution (GAMBROSOL BIO) Gambro 23/12/1999 For the treatment of patients receiving peritoneal dialysis who may need to change from peritoneal dialysis because of decreased ultrafiltration or infusion pain.
Peritoneal dialysis solution with 1.1% amino acids (NUTRINEAL-PD4) Baxter Health Care Pty Ltd 04/03/1999 For the treatment of protein malnutrition in patients with End Stage Renal Disease (ESRD) who are maintained by peritoneal dialysis.
PHYSIONEAL 40 brand of peritoneal dialysis solutions containing 13.6mg/mL, 22.7mg/mL or 38.7mg/mL glucose Baxter Healthcare Pty Ltd 08/05/2002 11/05/2005 For peritoneal dialysis.
Pertuzumab Roche Products Pty Limited 19/01/2012 For the treatment of patients with HER-2 positive metastatic (Stage IV) or locally recurrent breast cancer. Concentrate for intravenous infusion
Piracetam (NOOTROPIL) UCB Pharma 04/05/2000 as "add-on" treatment of cortical myoclonus.
Plerixafor (MOZOBIL) Genzyme Australasia Pty Ltd 20/10/2008 For the mobilisation of haematopoietic stem cells to the peripheral blood for collection and subsequent transplantation. Injection
Pomalidomide Celgene Pty Ltd 17/10/2012 For the treatment of multiple myeloma in patients who have failed two or more prior therapies. Capsules
Ponatinib (tradename TBA) ARIAD Pharmaceutical (Australia) Pty Ltd 14/05/2013 For the treatment of acute lymphoblastic leukaemia and adult patients with chronic phase, accelerated phase, or blast phase chronic myeloid leukaemia (CML) that is resistant or intolerant to prior tyrosine kinase inhibitor (TKI) therapy. 15mg and 45mg immediate film coated tablets
Poractant alfa (CUROSURF) Douglas Pharmaceuticals Australia Ltd 11/04/2003 13/01/2005 For the treatment of Respiratory Distress Syndrome (RDS) in preterm infants and for prophylactic use in preterm infants at risk for RDS.
Pralatrexate (FOLOTYN) Mundipharma Pty Limited 05/09/2011 For the treatment of adult patients with relapsed or refractory peripheral T-cell lymphoma (nodal, extranodal and leukaemic/disseminated). Solution for Injection
Pralidoxime chloride Ophthalmic Laboratories Pty Ltd (T/A Pharmalab) 03/05/2006 For the treatment of poisoning due to those pesticides and chemicals of the organophosphate class which have anticholinesterase activity and the control of overdosage of the anticholinesterase drugs used in the treatment of myasthenia gravis.
Propranolol hydrochloride Pierre Fabre Medicament Australia Pty Limited 03/10/2012 For the treatment of proliferating infantile hemangiomas requiring systemic therapy. Oral solution

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Q - Orphan drugs

There are no orphan drugs beginning with 'q'.

R - Orphan drugs

Hint: Click on the table headers to sort the table by a particular column in ascending or descending order.

Drug Sponsor Date of designation Date registered in Australia Indication Dose
Rabies immunoglobulin Pasteurised (IMOGAM RAGE) CSL 16/10/1998 24/02/2000 To treat subjects who are thought to have been exposed to rabies virus, especially cases of major exposure, in accordance with WHO recommendations.
Raltegravir (ISENTRESS) Merck Sharp & Dohme (Australia) Pty Limited 05/09/2011 Treatment of HIV-1 infection in children and adolescents from 2 to 18 years of age. Tablet
Ramucirumab (Cyramza) Eli Lilly Australia Pty Limited 17/09/2013 The indication is for the treatment of patients with advanced gastric cancer or gastro-oesophageal junction adenocarcinoma after prior chemotherapy. Solution for infusion
Ranpirnase (ONCONASE) Delpharm Consultants Pty Ltd 22/03/2005 For the treatment of malignant mesothelioma.
Rasburicase (FASTURTEC) Sanofi-Synthelabo Aust Pty Ltd 23/03/2001 15/07/2002 For the treatment and prophylaxis of acute hyperuricaemia in patients with haematological malignancy at risk of a rapid tumour lysis.
Recombinant factor IX (BENEFIX) Wyeth 16/10/1998 12/10/2000 For the control and prevention of haemorrhagic episodes and for routine and surgical prophylaxis in patients with haemophilia B (congenital factor IX deficiency or Christmas disease).
Recombinant factor IX (FIXTERA) Baxter Healthcare Pty Ltd 03/10/2012 For the treatment of the control and prevention of haemorrhagic episodes and for routine and surgical prophylaxis in patients with haemophilia B (congenital factor IX deficiency or Christmas disease). IV injection
Recombinant factor VIII (ADVATE) Baxter Healthcare Pty Limited 10/03/2003 For the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency).
Recombinant factor VIII(rDNA)(cho) (REFACTO) Wyeth 18/01/1999 04/01/2001 For the control and prevention of haemorrhagic episodes and for routine and surgical prophylaxis in patients with haemophilia A (congenital factor VIII deficiency or classical haemophilia).
Recombinant human coagulation factor IX Fc fusion protein (rFIXFc) Biogen Idec Australia Pty Ltd 20/09/2012 For the treatment of the control and prevention of haemorrhagic episodes in patients with haemophilia B (congenital factor IX deficiency or Christmas disease), including the control and prevention of bleeding in surgical settings. Lyophilised powder for reconstitution IV injection
Recombinant human coagulation factor VIII Fc fusion protein Biogen Idec Australia Pty Ltd 22/02/2013   For the treatment of control and prevention (including routine prophylaxis) of bleeding episodes in adults and children with haemophilia A. Lyophilized powder for reconstitution IV injection
Recombinant human iduronate-2-sulfatase (IDURSULFASE) Genzyme Australia Pty Ltd 18/01/2006 For long-term treatment of patients with Hunter syndrome
Retroviral γc cDNA-containing vector (IMMUGENE) AVAX Australia Pty Ltd 21/08/2002

For the treatment of severe combined immunodeficiency X1 (SCID X1).

Status: The orphan drug designation for this product was withdrawn on 2 October 2002.

RidaForolimus (JENZYL) Merck Sharp & Dohme (Australia) Pty Ltd 14/09/2011 Treatment of metastatic soft tissue sarcoma or bone sarcoma. Gastro resistant tablet
Rifaximin (XIFAXAN) Norgine Pty Ltd 24/02/2011 For the prevention of the recurrence of Hepatic Encephalopathy in adults. Tablets
Riluzole (RILUTEK) Aventis Pharma 03/05/2001 For the treatment of amyotrophic lateral sclerosis (ALS).
Riociguat Bayer Australia Limited 16/10/2012 For the treatment of Pulmonary Arterial Hypertension (PAH) and Chronic Thromboembolic Pulmonary Hypertension (CTEPH) Immediate - release tablets
Rituximab (MabThera) Roche Products Pty Limited 13/10/2011 Induction of remission in patients with severely active anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis. Intravenous infusion
Romidepsin (ISTODAX) Celgene Pty Ltd 27/08/2010

For the treatment of:

  • patients with peripheral T-cell lymphoma (PTCL) who have received at least one prior systemic therapy; and
  • patients with cutaneous T-cell lymphoma (CTCL) who have received at least one prior systemic therapy.
10 mg powder for injection
Ruxolitinib (JAKAVI) Novartis Pharmaceuticals Australia Pty Limited 23/04/2012 For the treatment of primary myelofibrosis, post-polycythaemia vera myelofibrosis and post-essential thrombocythaemia myelobfibrosis. Tablets

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S - Orphan drugs

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Drug Sponsor Date of designation Date registered in Australia Indication Dose
Sapropterin dihydrochloride Serono Pty Ltd 30/08/2006 For treatment of patients with hyperphenylalaninemia. Oral tablet
Sirolimus Wyeth Australia Pty Limited 26/03/2007 For the prevention of coronary allograft vasculopathy. Tablets 1 mg, 2 mg and 5 mg and oral solution (1 mg/mL, 60 mL)
Somatropin (HUMATROPE PENFIL) Aza Research 10/06/1999 01/03/2001 For the treatment of growth retardation in pre-pubertal children with chronic renal insufficiency.
Somatropin (SAIZEN) Merck Serono Australia Pty Ltd 05/09/2011 Growth disturbance in pre-pubertal children with chronic renal insufficiency.
Somatropin (rbe) (GENOTROPIN) Pharmacia-Upjohn 08/05/2000 11/08/2003 For improvement of body composition and treatment of short stature in patients with Prader-Wili syndrome.
Somatropin (rbe) recombinant human growth hormone (GENOTROPIN) Pharmacia Australia Pty Limited 28/11/2001 13/06/2003 For treatment of growth disturbance in pre-pubertal children with chronic renal insufficiency.
Somatropin (rbe) recombinant human growth hormone (GENOTROPIN) Pharmacia Australia Pty Limited 02/11/2001 For treatment of growth failure children born small for gestational age.
Somatropin (rbe) recombinant human growth hormone (NORDITROPIN, NORDITROPIN PENSET 12 and 14, NORDITROPIN SIMPLEXX) Novo Nordisk Pharmaceuticals P/L 11/03/2002 20/01/2004 For treatment of severe growth failure due to intrauterine growth retardation (i.e. children born small for gestational age without spontaneous catch up growth by 2 years of age and height below -3SD at start of treatment).
Somatropin (rbe) recombinant human growth hormone (NORDITROPIN, NORDITROPIN SIMPLEXX) Novo Nordisk Pharmaceuticals P/L 22/05/2002 For treatment of severe growth failure due to intrauterine growth retardation (i.e. children born small for gestational age without spontaneous catch up growth by 2 years of age).
Somatropin (rbe) (NORDITROPIN SIMPLEXX and NORDITROPIN NORDIFLEX) Novo Nordisk Pharmaceuticals Pty Ltd 08/05/2009 For the treatment of prader-willi syndrome, for improvement of growth and body composition. Injection-solution multidose cartridges
Sorafenib (NEXAVAR) Bayer Australia Limited 04/03/2013   For the treatment of patients with radioactive iodine treatment (RAI) refractory, locally advanced or metastatic differentiated thyroid cancer (DTC). Tablet
Stay Safe Balance and Sleep Safe Balance range of sterile peritoneal dialysis solutions Fresenius Medical Care Australia Pty Ltd 21/08/2002 13/06/2003 For the management of end stage renal disease and acute renal failure.
Sunitinib malate (SUTENT) Pfizer Australia Pty Ltd 04/05/2010 For the treatment of pancreatic neuroendocrine tumours (pNET). 12.5 mg, 25 mg, 37.5 mg and 50 mg capsules

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T - Orphan drugs

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Drug Sponsor Date of designation Date registered in Australia Indication Dose
Tacrolimus (PROGRAF) Janssen-Cilag Pty Ltd 22/10/2003 For use as an immunosuppressant in lung transplantation.
Tacrolimus (PROGRAF) Janssen-Cilag Pty Ltd 06/09/2006 For use as an immunosuppressant in heart transplantation. 0.5, 1 and 5 mg capsules and a 5 mg/mL concentrate for intravenous infusion
Tadalafil (ADCIRCA) Eli Lilly Australia Pty Ltd 21/04/2010 For the treatment of pulmonary arterial hypertension (WHO Group I) to improve exercise ability. 20mg tablets
Tafamidis meglumine (VYMDAQEL) Pfizer Australia Pty Ltd 22/07/2011 For the oral treatment of Transthyretin Amyloidosis in adult patients with Symptomatic Polyneuropathy. 20mg soft gelatine capsules
Taliglucerase (ELELYSO) Pfizer Australia Pty Ltd 11/03/2011 For the treatment of Gaucher's Disease Powder for injection
Temozolomide (TEMODAL) Schering-Plough Pty Limited 24/07/2003

For treatment of:

  • recurrence of anaplastic astrocytoma and glioblastoma multiforme in children, following standard therapy
  • metastatic malignant melanoma in children.
Temozolomide (TEMODAL) Schering-Plough Pty Limited 21/09/2004 For the treatment of patients with newly diagnosed anaplastic astrocytoma and gioblastoma multiforme.
Temsirolimus (TORISEL) Wyeth Australia Pty Limited 17/03/2008 For the treatment of patients with mantle cell lymphoma. Concentrate for injection
Tenofovir disoproxil fumarate (VIREAD) Gilead Sciences Pty Ltd 13/10/2009 In combination with other antiretroviral agents for the treatment of HIV-infected patients 2 to 17 years of age. Oral powder (40mg/1 g) and tablet (300mg)
Terlipressin (GLYPRESSIN) Ferring Pharmaceuticals Pty Ltd 21/01/2004 (freeze dried powder with diluent) & 7/10/2009 (solution) For the treatment of bleeding oesophageal varices and for the treatment of hepatorenal syndrome.
  • Freeze dried powder (1 mg/vial) with diluent (5 mL/ ampoule) and;
  • solution (0.12 mg/ mL ampoule)
Terlipressin acetate (LUCASSIN) Ikaria Australia Pty Ltd 25/06/2010

For the treatment of:

  • patients with hepatorenal syndrome type 1 (HRS-1);and
  • oesophageal variceal haemorrhage (OVH).
Powder for injection
Thalidomide (THALOMID) Pharmion Pty Ltd 13/03/2002 03/10/2003

For the treatment of:

  • erythema nodosum leprosum or Type II lepra reactions; and
  • relapsing or refractory multiple myeloma.
Thalidomide (THALIDOMIDE PHARMION) Celgene Pty Ltd 10/10/2008 For the treatment of relapsing or refractory multiple myeloma and erythema nodosum leprosum or Type II lepra reactions. 50mg hard capsules
Thymoglobuline (rabbit anti-human thymcoyte immunoglobulin) Genzyme Australasia Pty Ltd 22/02/2007

For the treatment of:

  • prophylaxis and treatment of graft rejection in renal transplantation;
  • prophylaxis of graft rejection in heart transplantation and;
  • aplastic anaemia.
Powder for injection
Thyrotropin alfa (THYROGEN) Genzyme Aust 07/10/1999 17/06/2004 For use with radioactive iodine imaging and serum thyroglobulin (Tg) testing, undertaken for the detection of thyroid remnants and well-differentiated thyroid cancer in post-thyroidectomy patients maintained on hormone suppression therapy and at risk of recurrence.
Thyroptropin alfa-rch powder for injection Genzyme Australasia Pty Ltd 24/01/2005 For therapeutic use in post-thyroidectomy patients maintained on hormone suppression therapy in the ablation of thyroid remnant tissue in combination with radioactive iodine.
Tipifarnib (ZARNESTRA) Janssen-Cilag Pty Ltd 17/11/2004 For the treatment of acute myeloid leukaemia.
Tobramycin (TOBI) Novartis Pharmaceuticals Pty Ltd 08/11/2010 For the treatment of cystic fibrosis patients infected with Pseudomonas aeruginosa. Powder for inhalation
Tobramycin (TOBI)solution for inhalation Pathogenesis Corporation 29/09/1998 15/02/2000 For the treatment of cystic fibrosis patients infected with Pseudomonas aeruginosa.
Tobramycin sulfate Ophthalmic Laboratories Pty Limited (T/A Pharmalab) 28/04/2006 For treatment of patients with cystic fibrosis infected with Pseudomonas species.
Tobramycin sulfate solution for inhalation Ophthalmic Laboratories Pty Limited 18/06/2004 For the treatment of patients with cystic fibrosis infected with Pseudomonas species.
Tracleer (BOSENTAN) Actilion Pharmaceuticals Pty Ltd 04/10/2005 For the treatment of Primary Pulmonary Arterial Hypertension (WHO Class PH I) in patients with WHO Functional Class III or IV symptoms.
Trametinib GlaxoSmithKline 01/06/2012 For the treatment of the patients with BRAF V600 mutation positive unresectable or metastatic (Stage IV) melanoma. Tablets
Trastuzumab emtansine (RCH) Roche Products Pty Limited 02/05/2012 For the treatment of HER-2 positive breast cancer patients who have locally recurrent, locally advanced or metastatic (Stage IV) disease. Powder
Treprostinil (REMODULIN) (incorrectly gazetted as epoprostenol) Orphan Australia Pty Ltd 29/05/2001 20/05/2004 For the treatment of pulmonary arterial hypertension.
Tumour necrosis factor alpha 1-a (BEROMUN) Boehringer Ingelheim 21/10/1998 15/03/2001 As an adjunct to surgery for subsequent removal of the tumour (soft tissue sarcoma) so as to delay amputation, or in the palliative situation, for irresectable soft-tissue sarcoma of the limbs, used in conjunction with melphalan in hyperthermic isolated limb perfusion.
Turoctocog alpha (RCH) Novo Nordisk Pharmaceuticals Pty Ltd 04/09/2012 For the treatment and prophylaxis of bleeding episodes in patients with haemophilia A. Powder for solution for injection.

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U - Orphan drugs

There are no orphan drugs beginning with 'u'.

V - Orphan drugs

Hint: Click on the table headers to sort the table by a particular column in ascending or descending order.

Drug Sponsor Date of designation Date registered in Australia Indication Dose
Vandetanib (brand name yet to be advised) AstraZeneca Pty Ltd 15/09/2010 For the treatment of patients with unresectable locally advanced or metastatic medullary thyroid cancer. Tablets
Velaglucerase alfa Shire Australia Pty Ltd 08/12/2009 For the treatment of Gaucher disease. Powder for solution for injection
Vemurafenib Roche Products Pty Ltd 14/04/2011 For the treatment of unresectable Stage IIIC or Stage IV metastatic melanoma whose tumours harbour the BRAF mutation. Tablets
Vorinostat (ZOLINZA) Merck Sharp & Dohme (Australia) Pty Ltd 21/10/2008 For the treatment of patients with advanced cutaneous T-cell lymphoma (CTCL) who have progressive, persistent or recurrent disease subsequent to prior systemic therapy. Capsules
VX-770 Voisin Consulting Australia Pty Ltd 15/06/2010 For improving pulmonary function in cystic fibrosis patients with at least one allele with a G551D-CF transmembrane conductance regulator gene (CFTR) mutation or homozygous for F508del-CFTR mutation. 100 mg and 150 mg film coated tablet

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W - Orphan drugs

There are no orphan drugs beginning with 'w'.

X - Orphan drugs

There are no orphan drugs beginning with 'x'.

Y - Orphan drugs

There are no orphan drugs beginning with 'y'.

Z - Orphan drugs

Hint: Click on the table headers to sort the table by a particular column in ascending or descending order.

Drug Sponsor Date of designation Date registered in Australia Indication Dose
Zanolimumab Merck Serono Australia Pty Ltd 10/07/2007 For the treatment of patients with cutaneous T cell lymphoma. Solution for infusion at 20 mg/mL
Zemaira Brand of alpha proteinase inhibitor (Human) CSL Ltd 08/08/2007 For the treatment of chronic intravenous augmentation and maintenance therapy in individuals with alpha1-proteinase inhibitor deficiency and clinical evidence of emphysema. Powder for injection
Zoledronic acid (ZOMETA) Novartis Pharmaceuticals Australia Pty Ltd 08/05/2009 For the treatment of paediatric patients with severe osteogenesis imperfecta (OI). 4 mg powder for injection and 4 mg/5 mL concentrated injection

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Web page last updated: Monday, 7 April 2014

URL: http://www.tga.gov.au/industry/pm-orphan-drugs.htm