General AML

The FDA grants Venclexta® Breakthrough Therapy Designation for the treatment of AML

On 28th July 2017, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) to Venclexta® (venetoclax), a BCL-2 oral inhibitor, in combination with Low Dose cytarabine (LDAC) for the treatment of elderly patients with previously untreated Acute Myeloid Leukemia (AML) who are ineligible for intensive chemotherapy.1

The BTD granted by the FDA was based on data from an ongoing phase I/II study (NCT02287233), which aimed to evaluate the safety and preliminary efficacy of Venclexa® at the RPD2 (600mg) in combination with LDAC in previously untreated elderly patients ≥65 years with AML. Results from the study showed that Venclexa® in combination with LDAC was tolerable and exhibited durable efficacy in elderly untreated AML patients. More results from this study are reported here.

Furthermore, one of the investigators in this study, Assoc. Prof. Andrew Wei, Alfred HospitalMonash University, Melbourne, Australia, spoke to the AGP about the design and findings of this study. Of which, he highlighted that Venclexa® plus LDAC combination was “extremely well tolerated” and “the treatment results were quite remarkable” in elderly AML patients. He added that the long term survival rate in patients were also “very promising”.

  1. Roche: FDA grants breakthrough therapy designation for Venclexta in acute myeloid leukaemia. 2017 Jul 28. [Accessed 2017 Jul 31].
  2. Wei A. et al. Updated safety and efficacy results of phase 1/2 study of venetoclax plus low-dose cytarabine in treatment-naïve acute myeloid leukemia patients aged ≥ 65 years and unfit for standard induction therapy. Abstract S473. 22nd Congress of the European Hematology Association; 2017 June 22–25; Madrid, Spain.
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