FDA Grants Breakthrough Therapy Designation for Venclexta in Combination With Azacitidine for the Treatment of Patients With Myelodysplastic Syndromes

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that Venclexta^® (venetoclax) in combination with azacitidine has been granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with previously untreated intermediate, high- and very high-risk myelodysplastic syndromes (MDS) based on the revised International Prognostic Scoring System (IPSS-R). MDS are a rare group of blood cancers that gradually affect the ability of the bone marrow to produce normal blood cells. This can lead to weakness, frequent infections, anemia and debilitating fatigue that can profoundly affect a person’s quality of life. In some cases, MDS can also progress into acute myeloid leukemia (AML). Every year in the United States, approximately 10,000 people are diagnosed with MDS, and the median survival for those with higher-risk MDS is approximately 18 months.

“Higher-risk MDS is associated with poor prognosis, reduced quality of life, and limited treatment options,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “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.”

This designation was granted based on interim results from the Phase Ib M15-531 study investigating Venclexta plus azacitidine in people with previously untreated, higher-risk MDS. BTD is designed to accelerate the development and review of medicines intended to treat serious or life-threatening conditions with preliminary evidence that indicates they may demonstrate a substantial improvement over existing therapies. This is the 38th BTD for Genentech’s portfolio of medicines, and the 11th designation for its hematology portfolio.

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