Australian approval for new leukaemia drug

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A new anti-cancer drug has been granted approval by the TGA, following equivalent approvals in the United States and European Union. 

Venetoclax, to be marketed as VENCLEXTA, has been approved for Australian patients with relapsed or refractory chronic lymphocytic leukaemia (CLL) with 17p deletion, a mutation that makes the disease relatively resistant to standard treatment options, as well as for patients with relapsed or refractory CLL for whom no other treatment options are available.

CLL is the most common type of leukaemia in Australia, with 1300 people diagnosed each year.

VENCLEXTA was discovered and developed with scientists from pharmaceutical companies AbbVie and Genentech, as part of an international collaboration with the Walter and Eliza Hall Institute.

Walter and Eliza Hall Institute of Medical Research director Professor Doug Hilton AO welcomed news of the drug’s approval, most importantly for patients with limited treatment options.

“The fact that Australians with hard-to-treat chronic lymphocytic leukaemia can now benefit from a drug like venetoclax demonstrates how critically important medical research is to the health of our community,” said Professor Hilton.

Professor Hilton applauded the team effort by Australian researchers and their international partners that preceded the approval. 

“TGA approval of venetoclax is a major milestone in a journey spanning decades of powerful and innovative research by teams of leading scientists, clinicians and entrepreneurs, including more than one hundred researchers at Melbourne’s Walter and Eliza Hall Institute of Medical Research,” he said. 

The timeline of discovery began at the Walter and Eliza Hall Institute in 1988, with the identification of BCL-2, a protein enabling cancer cells to survive. 

Professor Hilton said scientists worldwide had subsequently been trying to find a way to ‘hit’ BCL-2, in order to stop cancer cell survival.

“Like a lethal arrow, venetoclax flies straight to the heart of BCL-2,” added Professor Hilton. 

The first clinical trials for venetoclax started in Melbourne at the Institute’s Victorian Comprehensive Cancer Centre partners The Royal Melbourne Hospital and Peter MacCallum Cancer Centre and were led by Australian haematologists.

Professor Andrew Roberts, a clinical haematologist at The Royal Melbourne Hospital and cancer researcher at the Walter and Eliza Hall Institute and the University of Melbourne, said venetoclax was being combined now with other approved drugs and undergoing phase 2 and phase 3 clinical trials in other blood cancers. 

“The hope is that venetoclax, potentially in combination with other approved drugs, could benefit more patients including those with other hard-to-treat types of blood cancer,” said Professor Roberts.

“Ongoing research suggests that this drug will be very active against other cancers, so this milestone may just be the beginning,” he added.

Research on BCL-2 and therapies targeting it has been supported by NHMRC, National Institutes of Health, Leukemia and Lymphoma Society (USA), Cancer Council of Victoria, Leukaemia Foundation, Australian Cancer Research Foundation, Snowdome Foundation, Victorian Cancer Agency and a Victorian State Government Operational Infrastructure Support (OIS) grant.