FDA Grants Breakthrough Therapy Designation to Venetoclax for Higher Risk Myelodysplastic Syndromes


Venetoclax is already approved for the treatment of adults with chronic lymphocytic leukemia or small lymphocytic lymphoma, and in some adults with newly diagnosed acute myeloid leukemia.

Venetoclax (Venclexta, AbbVie) has been granted Breakthrough Therapy Designation by the FDA in combination with azacytidine for the potential treatment of adults with previously untreated intermediate-, high-, and very high-risk myelodysplastic syndromes.

Myelodysplastic syndromes are a group of diverse bone marrow disorders in which the marrow does not produce normal amounts of healthy blood cells. Patients with these syndromes may have symptoms such as infections, anemia, spontaneous bleeding, and easy bruising. Approximately 10,000 patients are diagnosed with myelodysplastic syndromes in the United States each year, and nearly one-third of patients will be diagnosed with acute myeloid leukemia (AML).

“[Myelodysplastic syndromes are] a devastating diagnosis. Not only does it have the potential to greatly impact patients’ quality of life, but 30% of patients will also progress to AML,” said Jalaja Potluri, MD, executive medical director of oncology with AbbVie, in a press release. “This Breakthrough Therapy Designation underscores the need for more treatment options for these patients and the utility of venetoclax to potentially treat different forms of blood cancer.”

Venetoclax is a first-in-class medication that selectively binds to and inhibits the B-cell lymphoma-2 protein, which prevents cancer cells from undergoing their natural death in some cancers. In the United States, it is approved for the treatment of adults with chronic lymphocytic leukemia or small lymphocytic lymphoma. It is also approved in combination with azacytidine, decitabine, or low-dose cytarabine in adults with newly diagnosed acute myeloid leukemia who are 75 years of age or older or who have other medical conditions preventing the use of standard chemotherapy.

Serious adverse effects (AEs) can include tumor lysis syndrome (TLS), and patients receiving venetoclax should drink plenty of water to reduce their risk of TLS. Other serious AEs include neutropenia and infections.

The breakthrough designation is supported by data from the phase 1b M15-531 study. Venetoclax is also being investigated in combination with azacytidine for the treatment of myelodysplastic syndromes in the phase 1b M15-522 study of patients with relapsed or refractory disease, and in the phase 3 randomized VERONA study of patients with newly diagnosed high-risk myelodysplastic syndromes.

Breakthrough Therapy Designation helps to expedite the development and review of medications to treat serious medical conditions. It is granted when preliminary clinical evidence suggests that the investigational therapy may demonstrate substantial improvements over currently available therapies.


Venetoclax (Venclexta) Granted US FDA Breakthrough Therapy Designation (BTD) in Higher Risk Myelodysplastic Syndrome (MDS). News release. Abbvie. July 21, 2021. Accessed July 21, 2021. https://news.abbvie.com/news/press-releases/venetoclax-venclexta-granted-us-fda-breakthrough-therapy-designation-btd-in-higher-risk-myelodysplastic-syndrome-mds.htm

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