Venetoclax Receives Breakthrough Therapy Designation for Treatment of Myelodysplastic Syndrome (MDS)


The FDA has granted a breakthrough therapy designation for venetoclax following interim results from the ongoing M15-531 trial.

The FDA has granted a breakthrough therapy designation (BTD) for venetoclax (Venclexta) in combination with azacytidine (Vidaza) for adult patients with previously untreated intermediate-, high-, and very high-risk myelodysplastic syndrome (MDS).

The BTD is based on the interim results from the ongoing phase Ib M15-531 (NCT02942290) study, in which the combination of venetoclax plus azacytidine has generated preliminary evidence that the therapy results in superior improvement over existing therapies.

“Higher-risk MDS is associated with poor prognosis, reduced quality of life, and limited treatment options,” said Levi Garraway, MD, PhD, Roche’s Chief Medical Officer and Head of Global Product Development, in a news release. Garraway expressed hope that the agent’s approval represents potentially improved treatment outcomes for this patient population.

The trial’s main objective is to ascertain the recommended phase 2 dose and dosing schedule of the venetoclax/azacytidine combination, as well as to assess the safety profile and pharmacokinetics of the therapy.

Venetoclax functions by selectively binding and inhibiting the B-cell lymphoma 2 (BCL-2) protein. It is a targeted medicine designed to eliminate cancer cells by limiting apoptosis, or the process in which BCL-2 accumulates and stops cancer cells from self-destructing.

This is the sixth breakthrough therapy designation that venetoclax has received for treatment of hematological cancers. It has also received BTDs for untreated chronic lymphocytic leukemia (CLL), 2 for relapsed or refractory CLL, 2 for previously untreated acute myeloid leukemia (AML), and 1 for MDS.

Roche stated in a new release that, “every year in the US, approximately 10,000 people are diagnosed with MDS and the median survival for those with higher-risk MDS is approximately 18 months.” It is hoped that the Ib M15-531 interim results and consequent BTD indicate that improved treatments for patients with higher-risk MDS are in development.


FDA grants breakthrough therapy designation for venclexta in combination with azacitidine for the treatment of patients with myelodysplastic syndromes. Roche. News release. July 21, 2021. Accessed July 21, 2021.

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