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Scheduling delegate's final decisions, January 2018
Scheduling medicines and poisons
The delegate considered an application from the Therapeutic Goods Administration (TGA) for the scheduling of nusinersen (as heptadecasodium), a new chemical entity (NCE) for a human therapeutic medicine.
Nusinersen is an antisense oligonucleotide (ASO) indicated for the treatment of spinal muscular atrophy, an autosomal recessive progressive neuromuscular disease caused by mutation or deletion of the survival motor neuron 1 (SMN1) gene on the q arm of chromosome 5. This results in a deficiency of SMN protein. The SMN2 gene, also present on the same chromosome, transcribes a similar but generally truncated SMN protein that is unstable and defective. Fully functioning SMN protein is essential for normal function of the anterior horn cell. Its deficiency results in progressive loss of skeletal muscle. Nusinersen promotes the transcription of a full length SMN protein.
|Molecular weight||7501.0 g/mol|
|Chemical names||Nusinersen (as heptadecasodium)|
Nusinersen (as heptadecasodium) is not specifically scheduled and is not captured by any entry 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 October 2017 (SUSMP No. 18)).
Nusinersen is not classified in New Zealand. Nusinersen is a prescription only medicine in Canada, the European Union and the United States of America.
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; and
- The TGA evaluation report.
- 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 insulin degludec in Schedule 4, with an implementation date of 1 February 2018.
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:
- Nusinersen is an NCE with no marketing experience in Australia.
- The benefits and risks of nusinersen have been considered and are outlined in the Product Information.
- Nusinersen should be prescribed under the supervision of medical practitioners with experience in the diagnosis and management of spinal muscular atrophy.
- Nusinersen is proposed for use in hospitals.
- The use of nusinersen has risks that may require clinical evaluation, intervention and monitoring by a medical practitioner.
- Labelling needs to comply with the requirements for an injectable prescription medicine.
- Nusinersen does not appear to produce dependency and the abuse potential appears to be low.