Designation notices

21 September 2017

What is designation?

Sponsors seeking access to the Priority review pathway and/or an Orphan drug fee waiver must first obtain formal designation from TGA prior to lodging a submission for registration. The designation process is based around eligibility criteria which are designed to ensure that only medicines providing the most benefit to patients are eligible.

The designation process will determine whether a medicine is eligible for the Priority review pathway or Orphan drug designation and is the first step towards registration on the Australian Register for Therapeutic Goods (ARTG). Granting of a designation does not mean that the medicine will be registered. After designation, the sponsor must lodge a submission for registration which will be subject to a rigorous evaluation process resulting in a decision on whether the medicine should be registered on the ARTG.

Priority review designation is a formal decision to accept a Priority review designation application under Section 25AAA of the Therapeutic Goods Act 1989.

Orphan drug designation is a formal decision to designate a medicine as an Orphan drug under regulation 16J of the Therapeutic Goods Regulations 1990.

Details of approved Priority review and Orphan drug designations from 1 July 2017 can be found below. For registration details of products approved for supply in Australia, please search the Australian Register of Therapeutic Goods.

Please note that multiple designations may be granted for a single sponsor and active ingredient. Each designation is listed in a separate row below.

Previous orphan drug program

Amendments to the Therapeutic Goods Regulations 1990 which came into effect on 1 July 2017 changed the eligibility criteria for Orphan drug designation. A transition period applies to orphan drugs designated under the previous orphan drug program (pre 1 July 2017). A list of orphan designations under the previous program is available.

Approved designations for prescription medicines

BioMarin Pharmaceuticals Australia Pty Ltd

Designation: Orphan drug

Status: Active

Effective date: 19/09/2017

Lapse date: 19/03/2018

Active ingredient(s): sapropterin Dihydrochloride (KUVAN)

Dosage form(s): Tablet

For the treatment of HPA in patients with PKU or BH4 deficiency.

Roche Products Pty Limited

Designation: Priority review

Status: Active

Effective date: 29/08/2017

Lapse date: 28/02/2018

Active ingredient(s): emicizumab (HEMLIBRA)

Dosage form(s): Injection solution

For routine prophylaxis to prevent bleeding or reduce the frequency of bleeding episodes in patients with haemophilia A (congenital factor Vlll deficiency) with factor Vlll inhibitors. HEMLIBRA can be used in all age groups.

Roche Products Pty Limited

Designation: Orphan drug

Status: Active

Effective date: 25/08/2017

Lapse date: 25/02/2018

Active ingredient(s): emicizumab (HEMLIBRA)

Dosage form(s): Injection solution

For routine prophylaxis in patients with haemophilia A (congenital factor Vlll deficiency)

Roche Products Pty Limited

Designation: Priority review

Status: Active

Effective date: 21/08/2017

Lapse date: 21/02/2018

Active ingredient(s): alectinib (ALECENSA)

Dosage form(s): Hard capsule blister pack

For the treatment of patients with anaplastic lymphoma kinase (ALK)-positive, locally advanced or metastatic non-small cell lung cancer (NSCLC).