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Part B - Final decisions on matters not referred to an expert advisory committee
5. New Chemical Entities - medicines for human therapeutic use
5.2 Tezacaftor
Delegate's final decision
Final decision:
The delegate has made a final decision to create a new Schedule 4 entry for tezacaftor in the Poisons Standard as follows:
Schedule 4 - New Entry
TEZACAFTOR.
Implementation date: 1 June 2018
Reasons:
The matters under subsection 52E (1) of the Therapeutic Goods Act 1989 considered relevant by the delegate include:
- the risks and benefits of the use of a substance
- It is a new chemical entity with no clinical or marketing experience in Australia.
- the purposes for which a substance is to be used and the extent of use of a substance
- Tezacaftor will be used in combination with ivacaftor for the treatment of cystic fibrosis in patients who have the genotype for mutations known to be responsive to tezcaftor/ivacaftor.
- Tezacaftor will be prescribed by physicians with expertise in cystic fibrosis.
- the toxicity of a substance
- There are no acute serious toxicities.
- the dosage, formulation, labelling, packaging and presentation of a substance
- Tezacaftor is an oral medicine.
- the potential for abuse of a substance
- Unlikely.
- any other matters that the Secretary considers necessary to protect public health
- Nil.
Scheduling proposal
The delegate considered an application from the Therapeutic Goods Administration (TGA) for the scheduling of tezacaftor, a new chemical entity (NCE) for a human therapeutic medicine.
Substance summary
Tezacaftor is a CFTR corrector that acts directly on CFTR to treat the underlying cause of CF by improving the cellular processing and trafficking of CFTR, thereby increasing the quantity of functional CFTR at the cell surface.
Tezacaftor/ivacaftor as an orphan drug is indicated for the treatment of patients with cystic fibrosis (CF) aged 12 years and older who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence.
| Property | Tezacaftor |
|---|---|
| Chemical structure |  |
| IUPAC name | 1-(2,2-difluoro-2H-1,3-benzodioxol-5-yl)-N-{1-[(2R)-2,3-dihydroxypropyl]-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1Hindol-5yl}cyclopropane-1-carboxamide |
| Empirical formula | C26H27F3N2O6 |
| Molecular weight | 520.5 g/mol |
Scheduling status
Tezacaftor is not specifically scheduled in the Standard for the Uniform Scheduling of Medicines and Poisons (SUSMP) - the Poisons Standard that was in effect at the time the decision was made (Poisons Standard March 2018 (SUSMP No. 20)).
International regulations
On 4 July 2014, the European Medicines Agency granted tezacaftor orphan drug designation for the treatment of cystic fibrosis.
Tezacaftor does not appear to be a scheduled substance in New Zealand, the USA or Canada.
Delegate's considerations
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 for orphan application (D17-3501202, D17-3501201); and
- The new drug application; (D17-3509315).
The delegate noted that currently there are no issues of concern that require additional control other than by inclusion in Schedule 4.