Orphan drugs

12 November 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.

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 is made, a sponsor should first seek orphan drug designation. Designated orphan drugs are eligible for a waiver of their application and evaluation fees, but only if designation is received prior to submitting an application to register an orphan drug on the Australian Register of Therapeutic Goods (ARTG).

Information on applications for orphan drug status can be found in the Applications for orphan drug designation.

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.

5

Drug Sponsor Date of designation Date registered in Australia Dose Indication
5-aminolevulinic acid hydrochloride (Gliolan) Specialised Therapeutics Australia Pty Ltd 30/03/12 Powder for oral solution 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.
5-azacytidine Pharmion Pty Ltd 02/09/04 For the treatment of myelodysplastic syndrome.

6

Drug Sponsor Date of designation Date registered in Australia Dose Indication
6-chloro-2, 3, 4, 9-tetrahydro-1H-carboxamide Siena Biotech Italy 11/03/11 For the treatment of Huntington's Disease.

A

Drug Sponsor Date of designation Date registered in Australia Dose Indication
Adalimumab (HUMIRA) Abbott Australasia Pty Ltd 16/01/12 Solution for injection 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.
Adalimumab (HUMIRA) AbbVie Pty Ltd 02/11/12 Solution for injection 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.
Adefovir dipivoxil Gilead Sciences Pty Ltd 12/08/05 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 30/10/08 10mg oral tablets For the treatment of chronic hepatitis B in adolescent patients (12 to <18 years of age).
Afamelanotide (SCENESSE) Clinuvel Pharmaceuticals Ltd 08/11/10 Subcutaneous implant For the treatment of erythropoietic porphyrias.
Afatinib Boehringer Ingelheim Pty Limited 01/06/12 Tablets For the treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with Epidermal Growth Factor Receptor (EGFR) mutation (s).
Agalsidase alfa (REPLAGAL) PPD Australia Pty Limited 20/10/00 17/05/02 For the long-term enzyme replacement therapy in patients with Fabry disease.
Agalsidase beta (FABRAZYME) Genzyme Australasia Pty Ltd 22/05/00 03/05/02 For long term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry Disease.
Aimspro (Goat Serum Derived Product) Daval Australia Pty Ltd 22/08/05 For treatment of Krabbe's Disease (Globoid Cell Leucodystrophy).
Albutrepenonacog Alfa CSL Behring Australia Pty Ltd 27/10/14 Injection For the treatment and prophylaxis of bleeding associated with congenital factor IX deficiency (haemophilia B).
Aldesleukin (PROLEUKIN) CSL Limited 16/10/98 24/05/00 For the treatment of metastatic renal cell carcinoma.
Aldesleukin (PROLEUKIN) CSL Limited 16/10/98 To treat metastatic melanoma.
Algucosidase alfa (MYOZYME) Genzyme Australasia Pty Ltd 08/09/03 Powder for concentrate for solution for infusion For the treatment of Pompe disease (glycogen storage disease type II).
Alpha-1-proteinase inhibitor (GLASSIA) Baxter Healthcare Pty Ltd 03/07/12 Solution for IV injection For the treatment of chronic augmentation and maintenance therapy in adults with emphysema due to congenital deficiency of alpha-1-antitrypsin.
Ambrisentan GlaxoSmithKline Australia Pty Ltd 05/07/07 Tablets 5 mg & 10 mg For the treatment of patients with pulmonary arterial hypertension (PAH) to improve exercise capacity, delay clinical worsening and decrease the symptoms of PAH.
Anagrelide hydrochloride (AGRYLIN) Orphan Australia Pty Ltd 16/10/98 23/11/99 For the treatment of essential thrombocytosis.
Anagrelide hydrochloride (Thromboreductin) Orphan Australia Pty Ltd 23/04/09 0.5mg and 1.0mg capsules For treatment of essential Thrombocythaemia in patients with high or intermediate risk of thrombosis or bleeding.
Anakinra (KINERET) Invida Australia Pty Ltd 20/12/11 Solution for injection For the treatment of active systemic onset juvenile idiopathic arthritis (SoJIA) in children.
Anakinra (KINERET) Invida Australia Pty Ltd 08/05/13 Solution, pre-filled syringe 100 mg / 0.67 mL 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)
Anti-H5N1 Clinical Network Services Pty Ltd 12/02/14 Solution for infusion Passive immunotherapy in case of post-exposure or suspected post-exposure to H5N1 avian influenza virus.
Arsenic trioxide Phebra Pty Ltd 24/02/11 Tablets and oral liquid For the treatment of acute promyelocytic leukaemia.
Arsenic trioxide injection Pharmalab Pty Limited 21/08/03 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 08/06/04 For the treatment of patients with acute promyelocytic leukaemia.
Artemether and lumefantrine (RIAMET) Novartis Pharmaceuticals Australia Pty Limited 27/10/99 05/07/02 For the treatment of children and adults with infections due toPlasmodium falciparum or mixed infections including P falciparum.
Artesunate injection Link Pharmaceuticals trading as Link Medical Products Pty Ltd 17/01/07 Solution for injection For the treatment of severe malaria including cerebral malaria and as a second line treatment in chloroquine resistant malaria cases.
Asfotase alfa Alexion Pharmaceuticals Australasia Pty Ltd 15/10/12 Subcutaneous injection For the treatment of hypophosphatasia (HPP).
Axitinib Pfizer Australia Pty Ltd 14/04/11 Tablets For the treatment of advanced renal cell carcinoma (RCC).
Azacytidine (VIDAZA) Celgene Pty Ltd 17/06/08 Powder for suspension for injection For the treatment of myelodysplastic syndrome.
Aztreonam lysine Gilead Sciences Pty Ltd 16/07/07 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 Control of gram-negative bacteria, particularly pseudomonas aeruginosa, in the respiratory tract of patients with cystic fibrosis.

B

Drug Sponsor Date of designation Date registered in Australia Dose Indication
Beclometasone dipropionate (ORBEC) Orphan Australia Pty Ltd 31/10/08 Oral capsules containing 1 mg immediate release beclometasone dipropionate tablet and 1 mg enteric-coated beclometasone dipropionate tablet 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.
Bevacizumab (AVASTIN) Roche Products Pty Ltd 13/01/09 A concentrated solution for injection - 100mg in 4 mLs and 400 mg in 16 mLs For the treatment of malignant glioma.
Bevacizumab (AVASTIN) Roche Products Pty Ltd 21/03/14 Injection concentrated For the treatment of persistant, recurrent of Stage IV carcinoma of the cervix.
BicaVera range of sterile peritoneal dialysis solutions Fresenius Medical Care Australia Pty Ltd 29/10/02 For use as a peritoneal dialysis solution in the management of end stage renal disease and acute renal failure.
Blinatumomab Amgen Australia Pty Ltd 20/11/13 Powder for reconstitution, Injection For the treatment of B-precursor acute lymphoblastic leukemia.
Bortezomib (VELCADE) Janssen-Cilag Pty Ltd 22/10/03 08/02/06 Powder for injection For the treatment of resistant or relapsed multiple myeloma.
Bortezomib (VELCADE) Janssen-Cilag Pty Ltd 19/08/14 Powder for injection For the treatment of patients with mantle cell lymphoma.
Bosentan Omnicare Clinical Research 12/02/01 12/11/02 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 Pty Ltd 27/08/10 62.5 mg and 125 mg tablets For the treatment of digital ulcers in patients with systemic sclerosis.
Bosutinib Pfizer Australia Pty Ltd 13/08/12 Tablets For the second or third line treatment of chronic myeloid leukaemia.
Brentuximab vedotin (ADCETRIS) Nycomed Pty Ltd 17/09/12 Powder for infusion For the treatment of: relapsed or refractory Hodgkin lymphoma (HL) and; systemic anaplastic large cell lymphoma (SALCL).
Busulfan (BUSULFEX) Orphan Australia Pty Ltd 12/10/05 13/11/07 For use in combination with other chemotherapeutic agents and/or radiotherapy as a conditioning regimen prior to haematopoietic progenitor cell transplantation.

C

Drug Sponsor Date of designation Date registered in Australia Dose Indication
C1 esterase inhibitor (BERINERT P) CSL Limited 10/04/07 Powder for injection For the treatment of hereditary angioedema.
C1 esterase inhibitor (CINRYZE) Cedarglen Investments Pty Ltd 15/10/10 Powder for reconstitution for intravenous administration 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.
Caffeine citrate injection Ophthalmic Laboratories Pty Limited 02/08/07 Solution for injection 40 mg/2mL For the short term treatment of apnoea of prematurity of gestational age 28 to less than 33 weeks.
Caffeine citrate oral solution Ophthalmic Laboratories Pty Limited 02/08/07 25mg/5mL For the short term treatment of apnoea of prematurity of gestational age 28 to less than 33 weeks.
Canakinumab (ILARIS) Novartis Pharmaceuticals Australia Pty Limited 01/05/13 Powder for Injection For the treatment of active Systemic Juvenile Idiopathic Arthritis (sJIA) in patients aged 2 years and older.
Caprine hyperimmune serum against HIV lysate (AIMSPRO) Daval Australia Pty Ltd 15/05/09 4.5mg total protein/ml liquid for subcutaneous injection For the treatment of amyotrophic lateral sclerosis.
Carboprost as the tropmethamine (HEMABATE) Pharmacia & Upjohn 16/10/98 07/06/01 For the treatment of post-partum haemorrhage due to uterine atony.
Carglumic acid (CARBAGLU™) Emerge Health Pty Ltd 13/03/12 Dispersible tablet 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.
Carmustine (GLIADEL) Orphan Australia Pty Ltd 25/09/03 27/10/04 For use as an adjunct to surgery to prolong survival in patients with malignant glioma.
Carmustine implants (GLIADEL wafers) Rhone-Poulenc Rorer Australia 16/10/98 29/12/00 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 22/11/11 Powder for injection Routine prophylaxis for bleeding in patients with congenital Factor XIII A subunit deficiency.
Catumaxomab (REMOVAB) Biotech Regulatory Solutions 15/09/10 Concentrate for solution for infusion For the treatment of patients with malignant ascites due to EpCAM-positive carcinomas
Celecoxib (CELEBREX) Searle 10/04/00 17/08/99 To reduce the number of adenomatous colorectal polyps in familial adenomatous polyposis (FAP), as an adjunct to usual care (eg endoscopic surveillance, surgery).
Ceritinib (ZYKADIA) Novartis Pharmaceuticals Australia Pty Ltd 01/09/14 Hard capsule For the treatment of patients with non-small cell lung cancer (NSCLC) that is anaplastic lymphoma kinase (ALK)-positive.
Cholic acid Orpharma Pty Ltd 25/07/13 oral capsules 50 mg and 250 mg For the treatment of inborn errors of bile acid synthesis responsive to treatment with cholic acid.
Cholic acid (ORPHACOL) Emerge Health 04/09/14 Capsule For the treatment of inborn errors of primary bile acid synthesis due to 3β-hydroxy-Δ5-C27-steroid oxidoreductase (3β-HSD) deficiency or Δ4-3-oxosteroid 5β-reductase (Δ4-3-oxoR) deficiency.
Cladribine (LITAK) Orphan Australia Pty Ltd 23/01/03 22/06/04 For the treatment of hairy cell leukaemia and lymphoplasmacytic lymphoma (Waldenstrom's macroglobulinaemia).
Clobazam (FRISIUM) Sanofi-Aventis Australia Pty Ltd 14/09/09 Tablet For the treatment of paediatric refractory epilepsy.
Clofarabine (EVOLTRA) Hospira Australia Pty Ltd 02/10/07 Concentrated solution for infusion 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.
Colistimethate sodium Phebra Pty Ltd 31/10/08 Sterile lyophilised powder 1MIU and 2MIU (colistimethate base) in single use vials for nebulisation. For treatment of pseudomonas aeruginosa infections in patients with cystic fibrosis.
Combivir GlaxoSmithKline Australia Pty Ltd 10/07/07 Tablets (containing lamivudine 150 mg & zidovudine 300 mg) For antiretroviral combination therapy for the treatment of Human Immunodeficiency Virus (HIV) infected children under 12 years of age.
Cyclosporin Delpharm Consultants Pty Ltd 17/11/04 For the prevention of graft rejection of lung transplants.
Cytarabine intrathecal injection (DEPOCYT) Orphan Australia Pty Ltd 07/01/04 28/06/06 50mg/5mL injection vial For the treatment of symptomatic neoplastic meningitis.

D

Drug Sponsor Date of designation Date registered in Australia Dose Indication
Dabrafenib GlaxoSmithKline Australia Pty Ltd 30/05/12 Capsules For the treatment of patients with BRAF V600 mutation positive unresectable or metastatic (Stage IV) melanoma.
Darunavir (PREZISTA) Janssen-Cilag Pty Ltd 22/08/08 75 mg, 150 mg, 300 mg and 600 mg tablet For the treatment of HIV infection in paediatric patients (children and adolescents aged 6 to <18 years).
Dasatinib (SPRYCEL) Bristol-Myers Squibb Pharmaceuticals Pty Ltd 12/10/05 09/01/07 Tablets 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).
Decitabine (DACOGEN) Janssen-Cilag Pty Ltd 16/10/12 Injection For the treatment of adult patients ≥65 years with newly diagnosed de novo or secondary acute myeloid leukaemia (AML).
Deferasirox (EXJADE) Novartis Pharmaceuticals Australia Pty Limited 24/09/04 30/06/06 Dispersible tablets For the treatment of chronic iron overload in patients with transfusion-dependent anaemias.
Deferiprone (FERRIPROX) Orphan Australia Pty Ltd 22/05/01 02/04/03 For the treatment of iron overload in patients with thalassaemia major, unwilling or unable to take desferrioxamine therapy.
Defibrotide Link Healthcare 12/11/13 Concentrate for solution for infusion For the treatment of hepatic veno-occlusive disease (VOD).
Denosumab (XGEVA) Amgen Australia Pty Ltd 27/06/14 Solution for injection For the treatment of hypercalacaemia of malignancy that is refractory to intravenous bisphosphonates.
Denosumab (XGEVA) Amgen Australia Pty Ltd 03/10/12 Injection For the treatment of patients with giant cell tumours of the bone.
DEXPRAMIPEXOLE Biogen Idec Australia Pty Ltd 25/10/11 Tablets For the treatment of amyotrophic lateral sclerosis (ALS).
Dornase alpha (PULMOZYME) Roche Products Pty Ltd 28/04/04 For the management of demonstrated respiratory complications in cystic fibrosis in children under the age of five years.
Drisapersen GlaxoSmithKline Australia Pty Ltd 10/12/13 Solution for injection For the treatment of patients with Duchenne muscular dystrophy (DMD) bearing certain mutations that are amenable to treatment with exon 51 skipping.
Duodopa (LEVODOPA / CARBIDOPA) Abbott Products Pty Ltd 16/12/10 20mg/mL and 5mg/mL intestinal gel For the treatment of advanced idiopathic Parkinson's disease with severe motor fluctuations and not responding to oral treatment.

E

Drug Sponsor Date of designation Date registered in Australia Dose Indication
Ecallantide (KALBITOR) Link Medical Products Pty Ltd 13/05/11 Solution for Injection For the treatment of patients with hereditary angioedema.
Eculizumab (rmc) (SOLIRIS) Alexion Pharmaceuticals Australasia Pty Ltd 05/09/11 Solution for intravenous infusion 300mg in 30 mL vial For the treatment of atypical haemolytic uraemic syndrome (aHUS).
Eculizumab (SOLIRIS) Alexion Pharmaceuticals Australasia Pty Ltd 26/10/12 Concentrated solution for IV infusion vial For the treatment of infection - associated haemolytic uraemic syndrome.
Eculizumab (SOLIRIS) Cedarglen Investments Pty Ltd 10/04/07 solution for injection E For the treatment of paroxysmal nocturnal haemoglobinuria.
Efavirenz (STOCRIN) Merck Sharp & Dohme (Australia) Pty Ltd 31/10/00 16/08/02 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 30/06/06 21/01/08 For the long term treatment of patients with Hunter syndrome (Mucopolysaccharidosis II, MPS II).
Eliglustat Sanofi-Aventis Australia Pty Ltd 02/08/13 Capsules The indication is for the treatment of long term treatment of adult patients with Gaucher disease type 1 (GD1)
Elosulfase-Alpha  BioMarin Pharmaceutical Australia Pty Ltd  05/07/13 Solution for infusion  For the treatment of mucopolysaccharidosis IV type A (Morquio A syndrome, MPS IVA). 
Eltrombopag (Revolade) GlaxoSmithKline Australia Pty Ltd 29/10/14 Powder for oral liquid For the treatment of chronic idiopathic thrombocytopaenic purpura (ITP).
Eltrombopag olamine (REVOLADE) GlaxoSmithKline Australia Pty Ltd 17/07/08 Tablet For the treatment of idiopathic thrombocytopenic purpura (ITP).
Epoprostenol (VELETRI) Actelion Pharmaceuticals Australia Pty Ltd 17/01/12 Lyophilized powder for reconstitution Treatment of pulmonary arterial hypertension.
Epoprostenol sodium (FLOLAN) Glaxo Wellcome Australia Ltd 06/12/99 19/09/01 For the long term treatment of primary pulmonary hypertension in New York Heart Association (NYHA) Class III and IV patients.
Epoprostenol sodium (FLOLAN) GlaxoSmithKline Australia Pty Ltd 05/04/06 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 14/01/05 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 14/04/11 Tablets For the treatment of advanced (Stage IIIB) or metastatic (Stage IV) non-small cell lung cancer (NSCLC) with activating EGFR mutations.
Ethyl eicosapentaenoate Orphan Australia Pty Ltd 15/07/04 For the treatment of Huntington's Disease.
Everolimus (AFINITOR) Novartis Pharmaceuticals Australia Pty Limited 17/07/08 5 mg and 10 mg tablets For the treatment of advanced renal cell carcinoma (RCC).
Everolimus (AFINITOR) Novartis Pharmaceuticals Australia Pty Limited 26/07/10 5 mg and 10 mg tablets For the treatment of tuberous sclerosis complex (TSC), including SEGA, angiomyolipoma and lymphangioleiomyomatosis (LAM) associated with TSC.
Ex vivo cultured adult human mesenchymal stem cells (PROCHYMAL) Delpharm Consultants Pty Ltd 08/11/10 Solution for intravenous infusion For the treatment of acute Graft versus Host Disease (aGvHD) in patients who have undergone Haematopoietic Stem Cell Transplantation (HSCT).
Extraneal brand of icodextrin 7.5% w/v peritoneal dialysis solution Baxter Healthcare Pty Ltd 26/09/01 17/10/02 For use as an alternative osmotic agent in dialysis solutions for long dwell exchange in patients treated with peritoneal dialysis.

F

Drug Sponsor Date of designation Date registered in Australia Dose Indication
Factor eight inhibitor bypassing activity (FEIBA VH) Baxter Healthcare Pty Ltd 20/05/04 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 Australia Limited 17/06/99 28/02/01 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 Capsules For the treatment of primary myelofibrosis, of post-polycythaemia vera myelofibrosis and of post-essential thrombocythaemia myelofibrosis.
FEIBA NF factor eight inhibitor bypassing fraction 500U powder For injection vial with diluent vial Baxter Healthcare Pty Ltd 22/06/04 13/12/06 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 (Australia) Pty Ltd 12/12/05 For treatment of chronic, non-infectious uveitis affecting the posterior segment of the eye.
Fomivirsen sodium (VITRAVENE) Ciba Vision Australia Pty Ltd 01/04/99 02/05/00 For the local treatment of cytomegalovirus (CMV) retinitis in patients with acquired immunodeficiency (AIDS).
Forodesine (FOSODINE) Mundipharma Pty Ltd 24/03/09 100mg tablets and 5 mg/mL solution for injection For the treatment of cutaneous T-cell lymphoma (CTCL).

G

Drug Sponsor Date of designation Date registered in Australia Dose Indication
G17DT (IMMUNOGEN) Kendle R & D Pty Ltd 20/11/03 For the treatment of gastric and pancreatic cancers.
Galsulfase rch (NAGLAZYME) Cedarglen Investments Pty Ltd 18/01/06 16/02/07 Solution for injection For long-term enzyme replacement therapy in the treatment of patients with mucopolysaccharidosis type VI (MPS VI).
Gemtuzumab ozogamicin (MYLOTARG) Wyeth Australia Pty Ltd 25/10/00 For the treatment of acute myeloid leukaemia.
GENOTROPIN brand of somatropin (rbe) Pfizer Australia Pty Ltd 09/02/07 Powder for injection For the treatment of growth disturbance in children with chronic renal insufficiency.

H

Drug Sponsor Date of designation Date registered in Australia Dose Indication
Histamine dihydrochloride FH Faulding and Co Limited 25/10/00 For the treatment of metatastic melanoma (stage IV).
Human fibrinogen (RIASTAP) CSL Limited 15/05/09 Powder for injection by intravenous infusion For the treatment of congenital fibrinogen deficiency.
Human hepatitis B immunoglobulin (HEPATECT CP) Bioregulatory Consulting 03/06/10 Solution for intravenous infusion Prophylaxis against re-infection of a transplanted liver in patients who carry the surface antigen of the hepatitis B virus.
Human hepatitis B immunoglobulin (HEPATECT CP) Link Medical Products Pty Ltd 27/08/09 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 Ltd 23/01/03 For the treatment of severe congenital protein C deficiency.
Humatrope brands of somatropin powder for subcutaneous or intramuscular injection Eli Lilly Australia Pty Ltd 13/06/02 For the treatment of growth failure in children born small for gestational age.

I

Drug Sponsor Date of designation Date registered in Australia Dose Indication
Ibuprofen (PEDEA) Emerge Health Pty Ltd 29/05/13 Solution for Injection For the treatment of haemodynamically significant patent ductus arteriosus in preterm newborn infants less than 34 weeks of gestational age.
Icatibant (FIRAZYR) Shire Australia Pty Ltd 10/10/13 Solution for injection Treatment of angiotensin-converting-enzyme inhibitor (ACE-1) induced angioedema.
Icatibant (FIRAZYR) Shire Australia Pty Ltd 12/02/09 30mg/3mL pre-filled syringe For the treatment of acute attacks of hereditary angioedema.
Icodextrin 7.5% (EXTRANEAL PERITONEAL DIALYSIS SOLUTION) Baxter Healthcare Pty Ltd 16/10/98 16/12/99 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 13/10/00 For the treatment of primary pulmonary hypertension.
Imatinib mesylate (GLIVEC) Novartis Australia Pty Ltd 12/05/11 Tablets and capsules For the treatment of pulmonary arterial hypertension.
Imatinib mesylate (GLIVEC) Novartis Pharmaceuticals Australia Pty Limited 12/08/02 28/02/03 For the treatment of chronic myeloid leukaemia.
Imatinib mesylate (GLIVEC) Novartis Pharmaceuticals Australia Pty Limited 10/12/01 21/06/02 For the treatment of adult patients with unresectable and/or metastatic malignant gastrointestinal stromal tumours.
Imatinib mesylate (GLIVEC) Novartis Pharmaceuticals Australia Pty Limited 01/10/08 5 mg & 100 mg capsules and 100 mg & 400 mg tablets For use as adjuvant therapy For gastrointestinal stromal tumour (GIST).
Imatinib mesylate (GLIVEC) Novartis Pharmaceuticals Australia Pty Limited 03/05/06 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 Australasia Pty Ltd 16/10/98 17/05/99 For enzyme replacement for patients with a confirmed diagnosis of Gaucher's disease.
Immunoglobulin - antithymocyte (rabbit) (ATG-Fresenius) Dutec Diagnostics Pty Ltd 18/06/10 Solution for intravenous injection For the prevention of graft versus host disease in allogeneic haematopoietic stem cell transplantation.
Indinavir sulfate (CRIXIVAN) Merck Sharp & Dohme (Australia) Pty Ltd 03/10/00 18/09/01 For the treatment of HIV-1 infection in children and adolescents over the age of 3 years.
Infliximab Schering-Plough Pty Ltd 12/10/05 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 Australia Pty Ltd 14/08/14 Tablets For the treatment of cystic fibrosis (CF) in patients who have a CFTR mRNA Splice Site (Class V) mutation or a residual function CFTR mutation in the CFTR gene.
Ivacaftor (KALYDECO) Vertex Pharmaceuticals Australia Pty Ltd 14/03/12 Tablets -film coated 150mg For the treatment of cystic fibrosis (CF) in patients who have a gating or an R117H mutation in the CFTR gene.
Ivacaftor (VX-770) Vertex Pharmaceuticals Australia Pty Ltd 22/09/14 Tablets In combination with Lumacaftor (VX-809) for the treatment of cystic fibrosis (CF) in patients who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

L

Drug Sponsor Date of designation Date registered in Australia Dose Indication
Lamivudine (ZEFFIX 3TCTM) Glaxo Wellcome Australia Ltd 11/01/01 18/12/01 For use in combination with other antiretroviral agents for the treatment of HIV infection in children.
Lamivudine (ZEFFIX) GlaxoSmithKline Australia Pty Ltd 19/06/02 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.
Lanreotide (SOMATULINE AUTOGEL) Ipsen Pty Ltd 19/03/04 17/12/04 For the treatment of the symptoms of carcinoid syndrome associated with carcinoid tumours and the symptoms of VIPomas.
Laronidase (ALDURAZYME) Genzyme Australasia Pty Ltd 08/08/01 04/05/04 For the treatment of mucopolysaccharidosis.
Leflunomide Aventis Pharma Pty Ltd 08/04/04 For treatment of polyarticular course juvenile rheumatoid arthritis in patient 3 to 17 years of age.
Lenalidomide (REVLIMID) Celgene Pty Ltd 06/09/06 13/12/07 Capsules 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.
Lepirudin (REFLUDAN) Celgene Pty Ltd 09/10/08 50mg powder for injection 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).
Lepirudin (REFLUDAN) Hoechst Marion Roussel 16/10/98 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 19/12/12 Injection For the treatment of Central Precocious Puberty (CPP).
Linezolid (ZYVOX) Pharmacia Australia Pty Ltd 09/01/03 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.
Lumacaftor (VX-809) Vertex Pharmaceuticals (Australia) Pty Ltd 22/09/14 Tablets In combination with Ivacaftor (VX-770) for the treatment of cystic fibrosis (CF) in patients who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

M

Drug Sponsor Date of designation Date registered in Australia Dose Indication
M-iodobenzyl [123I] guanidine injection (MIBGEN) Australian Nuclear Science and Technology Organisation (ANSTO) 15/08/02 26/05/04 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.
Macitentan Actelion Pharmaceuticals Australia Pty Ltd 13/07/12 Film coated tablets For the treatment of pulmonary arterial hypertension.
Mannitol (BRONCHITOL) Pharmaxis Ltd 16/04/09 40mg powder for inhalation For the treatment of patients with cystic fibrosis to improve lung function and reduce pulmonary exacerbations.
Mecasermin [rhIGF-1] injection (INCRELEX) 10mg/ml in 40 mg vial Ipsen Pty Ltd 01/07/08 Solution for subcutaneous injection For the long-term treatment of growth failure in children and adolescents with severe primary insulin-like growth factor-1 deficiency (severe primary IGFD).
Melphalan (ALKERAN INJECTION) Glaxo Wellcome Australia Ltd 22/03/99 04/09/01 For the treatment of multiple myeloma in patients For whom oral therapy is inappropriate.
Mepolizumab (BOSATRIA) GlaxoSmithKline Australia Pty Ltd 17/03/08 Powder for injection For the treatment of patients with hypereosinophilic syndrome
Mercaptopurine Ballia Holdings Pty Ltd 14/12/12 Oral suspension For the treatment of Acute Lymphoblastic Leukaemia in Children.
Mercaptopurine (XALUPRINE) Link Medical Products Pty Ltd 15/08/12 Oral suspension For the treatment of Acute Lymphoblastic Leukaemia in paediatric patients.
Methotrexate Ballia Holdings Pty Ltd 14/12/12 Oral suspension For the treatment of Acute Lymphoblastic Leukaemia in Children.
Midazolam (MIDAZOLAM BUCCAL LIQUID) Phebra Pty Ltd 01/06/09 10mg in 1mL liquid to be administered via an oral syringe into the buccal cavity For the treatment of status epilepticus in children and adolescents aged 0 to 19 years.
Mifamurtide (Mepact) Nycomed Pty Ltd 02/09/13 Powder for infusion
Miglustat (ZAVESCA) Actelion Pharmaceuticals Australia Pty Ltd 11/07/05 02/03/07 Capsules For the treatment of mild to moderate type 1 Gaucher disease.
Miglustat (ZAVESCA) Actelion Pharmaceuticals Australia Pty Ltd 08/07/08 Capsules For the treatment of Niemann-Pick disease Type C.
Mometasone furoate RhinoScience Pty Ltd 14/09/11 Irrigation base equivalent 2mg/240mL solution For the treatment of persistent post-surgical eosinophilic polypoid chronic rhinosinusitis.
MultiBic Fresenius Medical Care Australia Pty Ltd 16/08/04 For the treatment of acute renal failure in intensive care units.
Mycophenolic acid (as sodium) MYFORTIC Novartis Pharmaceuticals Australia Pty Limited 11/01/10 180 mg and 360 mg enteric coated tablets For induction and maintenance treatment of lupus nephritis.

N

Drug Sponsor Date of designation Date registered in Australia Dose Indication
Nandrolone decanoate (DECA-DURABOLIN 100) Organon 30/12/99 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 01/12/09 Powder for injection (suspension) For treatment of pancreatic cancer.
Natalizumab (TYSABRI) Nitecs Pty Limited 01/07/14 Concentrated injection solution For the treatment of relapsing-remitting multiple sclerosis in children aged 10-17 years.
Nevirapine (VIRAMUNE) Boehringer Ingelheim Pty Limited 11/03/03 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 Limited 09/06/06 14/01/08 200mg capsules blister packs For treatment of patients with chronic myeloid leukaemia.
Nitazoxanide (CRYPTAZ) Tri-Med Distributors 13/10/99 For the treatment of patients with AIDS who are positive for a stool test for cryptosporidiosis.
Nitisinone (ORFADIN) Orphan Australia Pty Ltd 30/10/08 2mg, 5mg and 10mg capsules For the treatment of hereditary tyrosinaemia type 1.
Nitric oxide (INOmax) Delpharm Consultants Pty Ltd 11/05/06 16/11/07 800 ppm medicinal gas for inhalation cylinder For the treatment of Persistent Pulmonary Hypertension (PPH) in neonates.
Nitric Oxide (INOmax) Ikaria Australia Pty Ltd 11/09/13 indication is inhalational gas 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.
Normal immunoglobulin (human) (KIOVIG) Baxter Healthcare Pty Ltd 29/11/12 Intravenous use injection For the treatment of immunomodulatory therapy in multifocal motor neuropathy (MMN).
Normal immunoglobulin (INTRAGLOBIN F) Medical Dynamics Australia (MDA) Pharma 27/02/01 11/04/01 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 30/01/07 Powder for injection 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.
NSC-631570 (UKRAIN) Mrs Aleksandra Harasemcuk 08/06/04 For the treatment of pancreatic cancer.

O

Drug Sponsor Date of designation Date registered in Australia Dose Indication
Oblimersen (GENASENSE) Aventis Pharma Pty Ltd 25/09/03 For the treatment of patients with stage IV malignant melanoma.
Oblimersen (GENASENSE) ICON Clinical Research 10/01/08 Solution for infusion For the treatment of patients with stage IV malignant melanoma.
Oblimersen (GENASENSE) Kendle R & D Pty Ltd 24/11/06 For the treatment of patients with stage IV malignant melanoma.
Octocog alfa rch (ADVATE) intravenous injection Baxter Healthcare Pty Ltd 19/02/07 05/12/07 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 10/03/04 25/02/05 Is indicated for use in haemophilia A for prevention and control of haemorrhagic episodes.
Octreotide (SANDOSTATIN / SANDOSTATIN LAR) Novartis Pharmaceuticals Australia Pty Limited 15/05/09 ·         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 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.
Octreotide (SANDOSTATIN LAR) Novartis Pharmaceuticals Australia Pty Limited 15/09/10 10, 20 and 30 mg prefilled syringe composite packs For the treatment of patients with advanced neuroendocrine tumours of the midgut or unknown primary tumour location.
Olaparib AstraZeneca Pty Ltd 15/01/14 Capsule 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.
Oprelvekin (IL-11) (NEUMEGA) Wyeth Australia Pty Ltd 07/04/99 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 04/07/11 Ampoules As an antidote in cases of acute colchinine toxicity.

P

Drug Sponsor Date of designation Date registered in Australia Dose Indication
Panobinostat (TBA) Novartis Pharmaceuticals Australia Pty Limited 24/03/09 Capsules For the treatment of cutaneous T-cell lymphoma (CTCL).
Pasireotide (SIGNIFOR LAR) Novartis Pharmaceuticals Australia Pty Limited 29/10/13 Modified release injection For the treatment of patients with Acromegaly for whom medical therapy is appropriate.
Pasireotide (SIGNIFor) Novartis Pharmaceuticals Australia Pty Limited 03/08/11 Solution for injection For the treatment of patients with Cushing's disease for whom medical therapy is appropriate.
Pazopanib (PATORMA) GlaxoSmithKline Australia Pty Ltd 24/03/09 200mg and 400mg film coated tablets For the treatment of advanced and/or metastatic renal cell carcinoma (RCC).
Pazopanib (VOTRIENT) GlaxoSmithKline Australia Pty Ltd 13/05/11 Tablets For the treatment of advanced (unresectable and/or metastatic) soft tissue sarcoma.
PEDITRACE and ADDAMEL N brands of trace element solutions for intravenous injection Fresenius Medical Care Australia Pty Ltd 08/11/01 For the supply of basal trace element requirements in infants/children and adults, respectively.
Pegaspargase (PEGASPAR) Rhone-Poulenc Rorer Australia 20/01/99 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 Ltd 15/06/12 Injection For the treatment of chronic hepatitis C in treatment-naïve children and adolescents 5 years of age and older with compensated liver disease.
Peginterferon alfa-2b and ribavirin (PEGATRON COMBINATION THERAPY) Schering-Plough Pty Ltd 15/01/10 Peginterferon alfa-2b; 50, 80, 100, 120 and 150 microgram powder for injection and ribavirin 200 mg capsule. For the treatment of chronic hepatitis C in children = 27 kg with compensated liver disease and who have not received previous interferon treatment.
Pegvisomant (rbe) (SOMAVERT) Pharmacia Australia Pty Ltd 09/10/03 07/12/05 Powder for injection with diluent For the treatment of patients with acromegaly.
Pegylated liposomal doxorubicin (CAELYX) Schering-Plough Pty Ltd 05/07/07 Injection vial 20 ml/10mL and 50mg/25mL 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.
Pegylated liposomal doxorubicin hydrochloride (CAELYX) Schering-Plough Pty Ltd 28/02/00 21/09/01 For the treatment of advanced ovarian cancer in women who have failed first-line platinum-based chemotherapy.
Pentostatin (NIPENT) Hospira Australia Pty Ltd 15/05/09 10mg powder for injection and powder for solution for infusion For the treatment of Hairy Cell Leukaemia (HCL)
Peritoneal dialysis solution (GAMBROSOL BIO) Gambro 23/12/99 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 Healthcare Pty Ltd 04/03/99 For the treatment of protein malnutrition in patients with End Stage Renal Disease (ESRD) who are maintained by peritoneal dialysis.
Pertuzumab Roche Products Pty Ltd 19/01/12 Concentrate for intravenous infusion For the treatment of patients with HER-2 positive metastatic (Stage IV) or locally recurrent breast cancer.
PHYSIONEAL 40 brand of peritoneal dialysis solutions containing 13.6mg/mL, 22.7mg/mL or 38.7mg/mL glucose Baxter Healthcare Pty Ltd 08/05/02 11/05/05 For peritoneal dialysis.
Piracetam (NOOTROPIL) UCB Pharma 04/05/00 as "add-on" treatment of cortical myoclonus.
Plerixafor (MOZOBIL) Genzyme Australasia Pty Ltd 20/10/08 Injection For the mobilisation of haematopoietic stem cells to the peripheral blood for collection and subsequent transplantation.
Pomalidomide Celgene Pty Ltd 17/10/12 Capsules For the treatment of multiple myeloma in patients who have failed two or more prior therapies.
Ponatinib (tradename TBA) ARIAD Pharmaceutical (Australia) Pty Ltd 14/05/13 15mg and 45mg immediate film coated tablets 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.
Poractant alfa (CUROSURF) Douglas Pharmaceuticals Australia Ltd 11/04/03 13/01/05 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 Ltd 05/09/11 Solution for Injection For the treatment of adult patients with relapsed or refractory peripheral T-cell lymphoma (nodal, extranodal and leukaemic/disseminated).
Pralidoxime chloride Ophthalmic Laboratories Pty Ltd (T/A Pharmalab) 03/05/06 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 Ltd 03/10/12 Oral solution For the treatment of proliferating infantile hemangiomas requiring systemic therapy.

R

Drug Sponsor Date of designation Date registered in Australia Dose Indication
Rabies immunoglobulin Pasteurised (IMOGAM RAGE) CSL Limited 16/10/98 24/02/00 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 Ltd 05/09/11 Tablet Treatment of HIV-1 infection in children and adolescents from 2 to 18 years of age.
Ramucirumab (Cyramza) Eli Lilly Australia Pty Ltd 17/09/13 Solution for infusion The indication is for the treatment of patients with advanced gastric cancer or gastro-oesophageal junction adenocarcinoma after prior chemotherapy.
Ranpirnase (ONCONASE) Delpharm Consultants Pty Ltd 22/03/05 For the treatment of malignant mesothelioma.
Rasburicase (FASTURTEC) Sanofi-Synthelabo Aust Pty Ltd 23/03/01 15/07/02 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 Australia Pty Ltd 16/10/98 12/10/00 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 03/10/12 IV injection 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).
Recombinant factor VIII (ADVATE) Baxter Healthcare Pty Ltd 10/03/03 For the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency).
Recombinant factor VIII(rDNA)(cho) (REFACTO) Wyeth Australia Pty Ltd 18/01/99 04/01/01 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 20/09/12 Lyophilised powder for reconstitution IV injection 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.
Recombinant human coagulation factor VIII Fc fusion protein Biogen Idec Australia Pty Ltd 22/02/13 Lyophilized powder for reconstitution IV injection For the treatment of control and prevention (including routine prophylaxis) of bleeding episodes in adults and children with haemophilia A.
Recombinant human iduronate-2-sulfatase (IDURSULFASE) Genzyme Australasia Pty Ltd 18/01/06 For long-term treatment of patients with Hunter syndrome
Retroviral γc cDNA-containing vector (IMMUGENE) AVAX Australia Pty Ltd 21/08/02 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 14/09/11 Gastro resistant tablet Treatment of metastatic soft tissue sarcoma or bone sarcoma.
Rifaximin (XIFAXAN) Norgine Pty Ltd 24/02/11 Tablets For the prevention of the recurrence of Hepatic Encephalopathy in adults.
Riluzole (RILUTEK) Aventis Pharma Pty Ltd 03/05/01 For the treatment of amyotrophic lateral sclerosis (ALS).
Riociguat Bayer Australia Limited 16/10/12 Immediate - release tablets For the treatment of Pulmonary Arterial Hypertension (PAH) and Chronic Thromboembolic Pulmonary Hypertension (CTEPH)
Rituximab (MabThera) Roche Products Pty Ltd 13/10/11 Intravenous infusion Induction of remission in patients with severely active anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis.
Romidepsin (ISTODAX) Celgene Pty Ltd 27/08/10 10 mg powder for injection 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.
Ruxolitinib (JAKAVI) Novartis Pharmaceuticals Australia Pty Ltd 16/10/14 Tablet For the treatment of patients with polycythaemia vera who are resistant to or intolerant of hydroxyurea.
Ruxolitinib (JAKAVI) Novartis Pharmaceuticals Australia Pty Limited 23/04/12 Tablets For the treatment of primary myelofibrosis, post-polycythaemia vera myelofibrosis and post-essential thrombocythaemia myelobfibrosis.

S

Drug Sponsor Date of designation Date registered in Australia Dose Indication
Sapropterin dihydrochloride Serono Pty Ltd 30/08/06 Oral tablet For treatment of patients with hyperphenylalaninemia.
Selexipag Actelion Pharmaceuticals Australia Pty Ltd 25/09/14 Tablet For the treatment of pulmonary arterial hypertension.
Siltuximab (Sylvant) Janssen-Cilag Pty Ltd 14/08/14 Powder for infusion Treatment of adults with multicentric Castleman's disease (MCD) who are human immunodeficiency virus (HIV) negative and human herpes virus-8 (HHV-8) negative.
Sirolimus Wyeth Australia Pty Ltd 26/03/07 Tablets 1 mg, 2 mg and 5 mg and oral solution (1 mg/mL, 60 mL) For the prevention of coronary allograft vasculopathy.
Sodium phenylbutrate (PHEBURANE) Orpharma Pty Ltd 13/08/14 Granules Treatment of urea cycle disorders.
Somatropin (HUMATROPE PENFIL) Aza Research 10/06/99 01/03/01 For the treatment of growth retardation in pre-pubertal children with chronic renal insufficiency.
Somatropin (rbe) (GENOTROPIN) Pharmacia & Upjohn 08/05/00 11/08/03 For improvement of body composition and treatment of short stature in patients with Prader-Wili syndrome.
Somatropin (rbe) (NORDITROPIN SIMPLEXX and NORDITROPIN NORDIFLEX) Novo Nordisk Pharmaceuticals Pty Ltd 08/05/09 Injection-solution multidose cartridges For the treatment of prader-willi syndrome, for improvement of growth and body composition.
Somatropin (rbe) recombinant human growth hormone (GENOTROPIN) Pharmacia Australia Pty Ltd 28/11/01 13/06/03 For treatment of growth disturbance in pre-pubertal children with chronic renal insufficiency.
Somatropin (rbe) recombinant human growth hormone (GENOTROPIN) Pharmacia Australia Pty Ltd 02/11/01 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 Pty Ltd 11/03/02 20/01/04 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 Pty Ltd 22/05/02 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 (SAIZEN) Merck Serono Australia Pty Ltd 05/09/11 Growth disturbance in pre-pubertal children with chronic renal insufficiency.
Sorafenib (NEXAVAR) Bayer Australia Limited 04/03/13 Tablet For the treatment of patients with radioactive iodine treatment (RAI) refractory, locally advanced or metastatic differentiated thyroid cancer (DTC).
Stay Safe Balance and Sleep Safe Balance range of sterile peritoneal dialysis solutions Fresenius Medical Care Australia Pty Ltd 21/08/02 13/06/03 For the management of end stage renal disease and acute renal failure.
Sunitinib malate (SUTENT) Pfizer Australia Pty Ltd 04/05/10 12.5 mg, 25 mg, 37.5 mg and 50 mg capsules For the treatment of pancreatic neuroendocrine tumours (pNET).
Susoctocog alfa Baxter Healthcare Pty Ltd 03/07/14 Powder for injection For the treatment and prevention of serious bleeding episodes in patients with acquired haemophilia A.

T

Drug Sponsor Date of designation Date registered in Australia Dose Indication
Tacrolimus (PROGRAF) Janssen-Cilag Pty Ltd 22/10/03 For use as an immunosuppressant in lung transplantation.
Tacrolimus (PROGRAF) Janssen-Cilag Pty Ltd 06/09/06 0.5, 1 and 5 mg capsules and a 5 mg/mL concentrate for intravenous infusion For use as an immunosuppressant in heart transplantation.
Tadalafil (ADCIRCA) Eli Lilly Australia Pty Ltd 21/04/10 20mg tablets For the treatment of pulmonary arterial hypertension (WHO Group I) to improve exercise ability.
Tafamidis meglumine (VYMDAQEL) Pfizer Australia Pty Ltd 22/07/11 20mg soft gelatine capsules For the oral treatment of Transthyretin Amyloidosis in adult patients with Symptomatic Polyneuropathy.
Taliglucerase (ELELYSO) Pfizer Australia Pty Ltd 11/03/11 Powder for injection For the treatment of Gaucher's Disease
Temozolomide (TEMODAL) Schering-Plough Pty Ltd 24/07/03 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 Ltd 21/09/04 For the treatment of patients with newly diagnosed anaplastic astrocytoma and gioblastoma multiforme.
Temsirolimus (TORISEL) Wyeth Australia Pty Ltd 17/03/08 Concentrate for injection For the treatment of patients with mantle cell lymphoma.
Tenofovir disoproxil fumarate (VIREAD) Gilead Sciences Pty Ltd 13/10/09 Oral powder (40mg/1 g) and tablet (300mg) In combination with other antiretroviral agents for the treatment of HIV-infected patients 2 to 17 years of age.
Terlipressin (GLYPRESSIN) Ferring Pharmaceuticals Pty Ltd 21/01/04 ·         Freeze dried powder (1 mg/vial) with diluent (5 mL/ ampoule) and; ·         solution (0.12 mg/ mL ampoule) For the treatment of bleeding oesophageal varices and for the treatment of hepatorenal syndrome.
Terlipressin acetate (LUCASSIN) Ikaria Australia Pty Ltd 25/06/10 Powder for injection For the treatment of: ·         patients with hepatorenal syndrome type 1 (HRS-1);and ·         oesophageal variceal haemorrhage (OVH).
Thalidomide (THALIDOMIDE PHARMION) Celgene Pty Ltd 10/10/08 50mg hard capsules For the treatment of relapsing or refractory multiple myeloma and erythema nodosum leprosum or Type II lepra reactions.
Thalidomide (THALOMID) Pharmion Pty Ltd 13/03/02 03/10/03 For the treatment of: ·         erythema nodosum leprosum or Type II lepra reactions; and ·         relapsing or refractory multiple myeloma.
Thymoglobuline (rabbit anti-human thymcoyte immunoglobulin) Genzyme Australasia Pty Ltd 22/02/07 Powder for injection For the treatment of: ·         prophylaxis and treatment of graft rejection in renal transplantation; ·         prophylaxis of graft rejection in heart transplantation and; ·         aplastic anaemia.
Thyroptropin alfa-rch powder for injection Genzyme Australasia Pty Ltd 24/01/05 For therapeutic use in post-thyroidectomy patients maintained on hormone suppression therapy in the ablation of thyroid remnant tissue in combination with radioactive iodine.
Thyrotropin alfa (THYROGEN) Genzyme Australasia Pty Ltd 07/10/99 17/06/04 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.
Tipifarnib (ZARNESTRA) Janssen-Cilag Pty Ltd 17/11/04 For the treatment of acute myeloid leukaemia.
Tobramycin (TOBI) Novartis Pharmaceuticals Australia Pty Limited 08/11/10 Powder for inhalation For the treatment of cystic fibrosis patients infected with Pseudomonas aeruginosa.
Tobramycin (TOBI)solution for inhalation Pathogenesis Corporation 29/09/98 15/02/00 For the treatment of cystic fibrosis patients infected with Pseudomonas aeruginosa.
Tobramycin sulfate Ophthalmic Laboratories Pty Ltd (T/A Pharmalab) 28/04/06 For treatment of patients with cystic fibrosis infected with Pseudomonas species.
Tobramycin sulfate solution for inhalation Ophthalmic Laboratories Pty Limited 18/06/04 For the treatment of patients with cystic fibrosis infected with Pseudomonas species.
Tracleer (BOSENTAN) Actelion Pharmaceuticals Australia Pty Ltd 04/10/05 For the treatment of Primary Pulmonary Arterial Hypertension (WHO Class PH I) in patients with WHO Functional Class III or IV symptoms.
Trametinib GlaxoSmithKline Australia Pty Ltd 01/06/12 Tablets For the treatment of the patients with BRAF V600 mutation positive unresectable or metastatic (Stage IV) melanoma.
Trastuzumab emtansine (RCH) Roche Products Pty Ltd 02/05/12 Powder For the treatment of HER-2 positive breast cancer patients who have locally recurrent, locally advanced or metastatic (Stage IV) disease.
Treprostinil (REMODULIN) (incorrectly gazetted as epoprostenol) Orphan Australia Pty Ltd 29/05/01 20/05/04 For the treatment of pulmonary arterial hypertension.
Tumour necrosis factor alpha 1-a (BEROMUN) Boehringer Ingleheim (Australia) Pty Ltd 21/10/98 15/03/01 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 04/09/12 Powder for solution for injection. For the treatment and prophylaxis of bleeding episodes in patients with haemophilia A.

V

Drug Sponsor Date of designation Date registered in Australia Dose Indication
Vandetanib (brand name yet to be advised) AstraZeneca Pty Ltd 15/09/10 Tablets For the treatment of patients with unresectable locally advanced or metastatic medullary thyroid cancer.
Velaglucerase alfa Shire Australia Pty Ltd 08/12/09 Powder for solution for injection For the treatment of Gaucher disease.
Vemurafenib Roche Products Pty Ltd 14/04/11 Tablets For the treatment of unresectable Stage IIIC or Stage IV metastatic melanoma whose tumours harbour the BRAF mutation.
Vorinostat (ZOLINZA) Merck Sharp & Dohme (Australia) Pty Ltd 21/10/08 Capsules For the treatment of patients with advanced cutaneous T-cell lymphoma (CTCL) who have progressive, persistent or recurrent disease subsequent to prior systemic therapy.
VX-770 Voisin Consulting Australia Pty Ltd 15/06/10 100 mg and 150 mg film coated tablet 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.

Z

Drug Sponsor Date of designation Date registered in Australia Dose Indication
Zanolimumab Merck Serono Australia Pty Ltd 10/07/07 Solution for infusion at 20 mg/mL For the treatment of patients with cutaneous T cell lymphoma.
Zemaira Brand of alpha proteinase inhibitor (Human) CSL Limited 08/08/07 Powder for injection For the treatment of chronic intravenous augmentation and maintenance therapy in individuals with alpha1-proteinase inhibitor deficiency and clinical evidence of emphysema.
Zoledronic acid (ZOMETA) Novartis Pharmaceuticals Australia Pty Limited 08/05/09 4 mg powder for injection and 4 mg/5 mL concentrated injection For the treatment of paediatric patients with severe osteogenesis imperfecta (OI).