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Scheduling delegate's final decisions, June 2017
Scheduling medicines and poisons
Part B - Final decisions on matters not referred to an expert advisory committee
New Chemical Entities – medicines for human therapeutic use
The delegate considered an application from the Therapeutic Goods Administration (TGA) for the scheduling of migalastat, a new chemical entity (NCE) for a human therapeutic medicine.
Migalastat acts as a pharmacological chaperone, binding to mutant forms of the enzyme, α-galactosidase A. Through binding, migalastat stabilises α-galactosidase A and allows it to be transported into lysosomes. Here, α-galactosidase A is able to reduce the levels of glycosphingolipids, fatty substances that can accumulate in cells in the body including in the heart and kidneys.
Migalastat is indicated for long-term treatment of adult and adolescent patients 16 years and older with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency) and who have an amenable mutation.
Migalastat is not specifically scheduled or captured by any group entries in the current Poisons Standard.
Migalastat is authorised in the EU. Migalastat is not classified in New Zealand, Canada or the USA.
The delegate decided to make a delegate-only decision. The Advisory Committee on Medicines Scheduling was not consulted.
The delegate considered the following in regards to this application for scheduling:
- Subsection 52E(1) of the Therapeutic Goods Act 1989;
- The Scheduling Policy Framework (2015) scheduling factors;
- The TGA evaluation report; and
- The new drug application.
The delegate noted that currently there are no issues of concern that require additional control other than by inclusion in Schedule 4.
Delegate's final decision
The delegate has made a final decision to amend the Poisons Standard to include Migalastat in Schedule 4, with an implementation date of 1 October 2017.
The delegate has decided that the wording for the schedule entry will be as follows:
Schedule 4 – New Entry
The delegate decided that the relevant matters under subsection 52E(1) of the Therapeutic Goods Act 1989 are: (a) the risks and benefits of the use of a substance; (b) the purpose and the extent of use of a substance; (c) the toxicity of a substance; (d) the dosage, formulation, labelling, packaging and presentation of a substance; and (e) the potential for abuse.
The delegate decided that the reasons for the final decision comprise the following:
- Migalastat is an NCE with no marketing experience in Australia.
- The proposed indication for Migalastat is for long-term treatment of adult and adolescent patients 16 years and older with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency) and who have an amenable mutation.
- Fabry disease is a rare disease associated with serious health risks. Treatment should be supervised by a specialist physician experienced in the diagnosis and management of Fabry disease.
- Treatment with Migalastat requires clinical evaluation and monitoring by a medical practitioner.
- Labelling needs to comply with the requirements for a prescription only medicine.
- The potential for abuse of Migalastat is low.