FDA Grants Venclexta/Onureg a Breakthrough Therapy Designation for Untreated MDS

The FDA will expedite the development and review of the drug combination to treat untreated intermediate- high- and very high-risk myelodysplastic syndrome.

The Food and Drug Administration (FDA) granted a breakthrough therapy designation to Venclexta (venetoclax) plus Onureg (azacitadine) for the treatment of patients with untreated intermediate-, high- and very high-risk myelodysplastic syndrome (MDS), according to Roche, the pharmaceutical company that is co-developing the drug with AbbVie.

A breakthrough therapy designation is given to drugs that treat serious or life-threatening illnesses and allows the FDA to speed up the development and review of the treatment. This breakthrough therapy designation was based off findings from the phase 1b M15-531 study.

M15-531 is a non-randomized, multicenter study with a goal to find the right dose of Venclexta plus Onureg for patients with MDS whose disease has not been treated before. The trial includes a dose-escalation portion and a safety expansion portion. Results will help guide dosing amounts for phase 2 of the trial.

The development of new therapies for this patient population is crucial, as about 10,000 people are diagnosed with MDS every year in the United States, and the average survival for those with higher-risk MDS is about 18 months. In other cases, MDS can progress into acute myeloid leukemia (AML), a more aggressive type of cancer of the bone marrow.

“Higher-risk MDS is associated with poor prognosis, reduced quality of life, and limited treatment options,” said Dr. Levi Garraway, Roche’s Chief Medical Officer and Head of Global Product Development, in a statement. “We are pleased that the FDA has granted Venclexta its sixth Breakthrough Therapy Designation in recognition of its potential to improve outcomes for people with MDS in combination with azacitidine.”

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