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Scheduling delegate's final decisions, January 2017

Scheduling medicines and poisons

16 January 2017

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2.13. Lumacaftor

Final decisions on matters not referred to an expert advisory committee

2. New Chemical Entities – medicines for human therapeutic use

2.13. Lumacaftor

Scheduling proposal

The delegate considered an application from the Therapeutic Goods Administration (TGA) for the scheduling of lumacaftor, a NCE for a human therapeutic medicine.

Substance summary

Lumacaftor is a selective CFTR corrector that acts on F508del-CFTR to increase the amount of functional CFTR at the cell surface to enhance chloride transport. Lumacaftor acts on CFTR to facilitate the cellular processing and trafficking of CFTR, allowing the protein to reach the cell surface, where it exhibits improved chloride channel function compared to uncorrected F508del-CFTR. F508del-CFTR that has been delivered to the cell surface by lumacaftor can be further potentiated by ivacaftor. Lumacaftor can increase the amount of normal CFTR at the cell surface and can correct certain other CFTR forms, including certain mutations that cause defects in processing. Ivacaftor has been shown to have CFTR potentiator properties.

Lumacaftor is a component of the FDC product ORKAMBI (lumacaftor/ivacaftor), which is indicated for the treatment of cystic fibrosis (CF) in patients age 12 years and older who are homozygous for the F508del mutation in the CFTR gene.

AAN – Lumacaftor.

Scheduling status

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

International regulations

Lumacaftor is not classified in New Zealand.

Delegate's consideration

The delegate made a delegate-only decision, hence 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; 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 lumacaftor in Schedule 4, with an implementation date of 1 February 2017.

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

Schedule 4 – New Entry

LUMACAFTOR.

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.

The delegate decided that the reasons for the final decision comprise the following:

  • It is a NCE with no clinical/marketing experience in Australia; and
  • The potential for abuse of lumacaftor is unlikely.

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