2.16. Venetoclax

Final decisions on matters not referred to an expert advisory committee

2. New Chemical Entities – medicines for human therapeutic use

2.16. Venetoclax

On this page: Scheduling proposal | Substance summary | Scheduling status | International regulations | Delegate's consideration | Delegate's final decision

Scheduling proposal

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

Substance summary

Venetoclax is a BH#-mimetic which acts as a B-cell lymphoma 2 (Bcl-2) inhibitor, blocking the anti-apoptotic Bcl-2 protein. This leads to programmed cell death of chronic lymphocytic leukemia (CCL) cells.

Venetoclax is indicated for patients with relapsed/refractory chronic lymphocytic leukaemia with 17p deletion and for patients without 17p deletion who have no other suitable treatment options.

Table 2.16: Identifiers, properties and naming of Venetoclax

Property	Venetoclax
CAS Number	1257044-40-8
Chemical structure
Molecular formula	C45H50ClN7O7S
Molecular weight	868.44 g/mol
Chemical name/s	4-(4-{[2-(4-Chlorophenyl)-4,4-dimethyl-1-cyclohexen-1-yl]methyl}-1-piperazinyl)-N-({3-nitro-4-[(tetrahydro-2H-pyran-4-ylmethyl)amino]phenyl}sulfonyl)-2-(1H-pyrrolo[2,3-b]pyridin-5-yloxy)benzamide (IUPAC)
ANN/INN	Venetoclax (ANN and INN)

Scheduling status

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

International regulations

Venetoclax is unclassified in New Zealand.

Venetoclax is a prescription medicine in Canada and the United States of America.

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;
• The advice of the Advisory Committee on Prescription Medicines; 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 Venetoclax 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:

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; and (c) the toxicity of a substance.

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

• Venetoclax is an NCE with no clinical or marketing experience in Australia.
• Venetoclax is indicated for patients with relapsed/refractory chronic lymphocytic leukaemia with 17p deletion and for patients without 17p deletion who have no other suitable treatment options.
• Venetoclax has reported risks of tumour lysis syndrome, neutropaenia, thrombocytpaenia, diarrhea, nausea, infections, autoimmune haemolytic anaemia and immune thryombocytopaenic purpura.
• The potential for abuse of Venetoclax is unlikely.