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Scheduling delegate's final decisions, March 2017

23 March 2017

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1.2 Sebelipase alfa_

Part B - Final decisions on matters not referred to an expert advisory committee

New Chemical Entities – medicines for human therapeutic use

1.2 Sebelipase alfa

Scheduling proposal

The delegate considered an application from the Therapeutic Goods Administration (TGA) for the scheduling of sebelipase alfa, a new chemical entity (NCE) for a human therapeutic medicine.

Substance summary

Sebelipase alfa is an enzyme replacement therapy that addresses the underlying cause of lysosomal acid lipase deficiency (LAL-D) by reducing substrate accumulation in the lysosomes of cells throughout the body.

Sebelipase alfa is indicated for long-term enzyme replacement therapy (ERT) in patients of all ages with lysosomal acid lipase deficiency (LAL-D).

Australian Approved Name (AAN) and International Non-Proprietary Name (INN): Sebelipase alfa

Scheduling status

Sebelipase alfa is not specifically scheduled and is not captured by any entry in the current Poisons Standard.

International regulations

Sebelipase alfa is not classified in New Zealand and Canada.

Sebelipase alfa is approved for use in the USA and the EU.

Delegate's consideration

The delegate decided to make a delegate-only decision. The Advisory Committee on Medicines Scheduling (ACMS) 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 Sebelipase alfa in Schedule 4, with an implementation date of 1 June 2017.

The delegate has decided that the wording for the schedule entry will be as follows:

Schedule 4 - New Entry

SEBELIPASE ALFA.

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:

  • It is an NCE with no clinical experience in Australia apart from clinical trials.
  • The risks and benefits of Sebelipase alfa have been considered and are outlined in the Product Information (PI), and the TGA evaluation reports.
  • Sebelipase alfa is indicated for long-term enzyme replacement therapy in patients with lysosomal acid lipase deficiency.
  • It has no previous experience of use in Australia outside the clinical trial setting but has recently been approved overseas.
  • Sebelipase alfa is a recombinant human lysosomal acid lipase given by intravenous infusion. Its use requires medical intervention, evaluation and monitoring by a medical practitioner.
  • Labelling needs to comply with the requirements for an injectable prescription only medicine.
  • It does not appear to produce dependency and the abuse potential appears low.

Book pagination