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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
5.7 Cerliponase alfa
Delegate's final decision
Final decision:
The delegate has made a final decision to create a new Schedule 4 entry for cerliponase alfa in the Poisons Standard as follows:
Schedule 4 - New Entry
CERLIPONASE ALFA.
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
- Cerliponase alfa is a new chemical entity with no clinical/marketing experience in Australia.
- the purposes for which a substance is to be used and the extent of use of a substance
- Cerliponase alfa is administered into the cerebral ventricles infusion via a surgically implanted intracerebroventricular (ICV) access device (reservoir and catheter).
- the toxicity of a substance
- Cerliponase alfa is for intra thecal use.
- the dosage, formulation, labelling, packaging and presentation of a substance
- Nil.
- the potential for abuse of a substance
- Nil.
- 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 cerliponase alfa, a new chemical entity (NCE) for a human therapeutic medicine.
Substance summary
Cerliponase alfa is an enzyme replacement therapy. Cerliponase alfa is a recombinant pro-enzyme of human tripeptidyl peptidase-1 (rhTPP1, also known as BMN 190). It is administered into the cerebral ventricles infusion via a surgically implanted intracerebroventricular (ICV) access device (reservoir and catheter). The ICV access device must be surgically implanted prior to initiating cerliponase alfa infusions. Cerliponase alfa and the flushing solution, which is administered immediately following the cerliponase alfa to ensure a complete delivery, are both administered via ICV infusion. Cerliponase alfa must only be administered by a trained healthcare professional knowledgeable in ICV administration in a healthcare setting.
Brineura is indicated for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency.
Scheduling status
Cerliponase alfa 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
Cerliponase alfa is classified as a prescription medicine in the USA and the EU.
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 (2018) scheduling factors; and
- 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.