Venclexta with Azacitidine Granted Breakthrough Therapy Designation for MDS

In a news release from July 21, 2021, biotechnology company Genentech shared that its therapy Venclexta (venetoclax), in conjunction with azacitidine, received Breakthrough Therapy designation from the FDA. Overall, the status was granted in regards to the treatment of adult patients with previously untreated intermediate, high, or very high-risk myelodysplastic syndromes (MDS).


So what exactly is Venclexta? This orally administered BLC-2 inhibitor is designed to stop the accumulation of BLC-2 protein. Normally, in certain cancers, BLC-2 can build up and prevent cancer cell death. Thus, by inhibiting this protein, Venclexta promotes cancerous cell death. Outside of this recent Breakthrough Therapy designation for MDS, Venclexta is prescribed for patients with chronic lymphocytic leukemia (CLL) or acute myeloid leukemia (AML).

Recently, Venclexta received its 6th Breakthrough Therapy designation from the FDA. According to the FDA, Breakthrough Therapy designation works to expedite drug development and review in cases where the treatment could improve patient outcomes or shows benefits over existing treatment options. In particular, the designation is granted to drugs which treat rare or life-threatening conditions. In this case, the designation hinged on data from the Phase 1b M15-531 clinical trial evaluating Venclexta and azacitidine for patients with MDS.

Venclexta Safety

Currently, Venclexta is approved for patients with CLL, small lymphocytic leukemia (SLL), and AML. Patients taking Venclexta should avoid grapefruit, Seville oranges, or starfruit. Additionally, patients should drink plenty of water while taking this treatment. Potential adverse reactions to Venclexta include:

  • Neutropenia (low white blood cell count)
  • Infertility (in males)
  • Thrombocytopenia (low platelet count)
  • Anemia (low red blood cell count)
  • Nausea and vomiting
  • Upper respiratory tract infections
  • Diarrhea or constipation
  • Muscle and joint pain
  • Cough
  • Extreme fatigue
  • Swelling of the hands, legs, and feet
  • Tumor lysis syndrome
  • Serious or life-threatening infections

Myelodysplastic Syndromes (MDS)

There is no known cause for myelodysplastic syndromes (MDS), progressive conditions that are often underserved and underdiagnosed. In patients with MDS, the bone marrow produces abnormal platelets, red blood cells, and white blood cells. These defective cells either die early, never leave the bone marrow, or are destroyed by the body. As a result, patients do not have enough healthy blood cells. Subtypes of MDS include refractory anemia, refractory anemia with sideroblasts, refractory anemia with excess blasts (or excess blasts in transformation), and chronic myelomonocytic leukemia.

Typically, MDS affects males slightly more than females. It also tends to impact older individuals (aged 60+), although it can affect people of any age. In around 50% of diagnoses, MDS progresses to become acute myeloid leukemia (AML). Sometimes, patients will not display any symptoms associated with MDS. However, if symptoms do appear, these include:

  • Anemia
  • Thrombocytopenia
  • Neutropenia
  • Fatigue and/or lethargy
  • Heart palpitations
  • Fever
  • Frequent infections
  • Easy bruising and bleeding
  • Shortness of breath
  • Chest pain
  • Mouth ulcers
  • Pallor (extremely pale skin)
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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