Policy & Regulation
FDA Grants Breakthrough Therapy Designation for Venclexta in Combination with Azacitidine for the Treatment of Patients with Myelodysplastic Syndromes
22 July 2021 - - US-based biotechnology company Genentech, a member of Switzerland's Roche Group (SIX: RO) (OTCQX: RHHBY), said that Venclexta (venetoclax) in combination with azacitidine has been granted Breakthrough Therapy Designation by the US Food and Drug Administration for the treatment of adult patients with previously untreated intermediate, high- and very high-risk myelodysplastic syndromes 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.

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.

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.

This most recent designation reinforces the potential of Venclexta-based combinations across several blood cancers, including MDS. In the United States, Venclexta has been granted six BTDs by the FDA: one for previously untreated chronic lymphocytic leukemia, two for relapsed or refractory CLL, two for previously untreated AML, and one for MDS.

Venclexta is already approved in the United States in combination with azacitidine, decitabine or low-dose cytarabine for the treatment of newly diagnosed AML in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy, and in the European Union in combination with hypomethylating agents, azacitidine and decitabine, for the treatment of adult patients with newly diagnosed AML who are ineligible for intensive chemotherapy.

Venclexta is also approved in the United States and European Union in combination with Rituxan (rituximab) for the treatment of adult patients with CLL who have received at least one prior therapy; in combination with Gazyva (obinutuzumab) for the treatment of adult patients with previously untreated CLL; and as a monotherapy for the treatment of CLL in the presence of 17p deletion or TP53 mutation in people who are unsuitable for or have failed a B-cell receptor pathway inhibitor.

Venclexta is being developed by AbbVie and Genentech, a member of the Roche Group. It is jointly commercialized by the companies in the United States and commercialized by AbbVie outside of the United States.
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