Mikkael Sekeres, MD @CleClinicMD @TakedaPharma FDA Breakthrough Therapy Designation Granted for Pevonedistat for the Treatment of Patients with Higher-Risk Myelodysplastic Syndromes (HR-MDS)
US. U.S. The Food and Drug Administration ( FDA) has awarded Breakthrough Therapy Designation for the treatment of patients with higher-risk myelodysplastic syndromes (HR-MDS) for its investigational drug pevonedistat. Pevonedistat, a first-in-class NEDD8-activating enzyme (NAE) inhibitor, may be the first innovative treatment in more than a decade for HR-MDS patients, extending treatment choices that have so far been limited to monotherapy alone with hypomethylating agent (HMA). Even with the latest care options, outcomes remain low for people living with HR-MDS.
The final review of the Pevonedistat-2001 Phase 2 trial, which evaluated pevonedistat + azacitidine versus azacitidine alone in patients with uncommon leukemias, including HR-MDS, is based on the Breakthrough Therapy Designation. A variety of endpoints, including overall survival ( OS), event-free survival (EFS), full remission (CR) and transfusion independence, were considered by the FDA, as well as the profile of adverse events. This classification represents a potential advance in meeting the needs of people living with HR-MDS, for whom there are few treatments and minimal benefits.
Designation of Breakthrough Therapy from the US The FDA is approved to speed up the production and regulatory approval of investigational drugs for the treatment of severe or life-threatening illnesses. Preliminary clinical data suggesting that the medication can demonstrate considerable improvement over existing therapies on one or more clinically relevant endpoints has been demonstrated by agents with this classification.