Posted by Michael Wonder on 30 Jul 2020
Takeda announces U.S. FDA breakthrough therapy designation granted for pevonedistat for the treatment of patients with higher-risk myelodysplastic syndromes
30 July 2020 - Designation based on Phase 2 data and reinforces significant treatment need for higher-risk myelodysplastic syndrome.
Takeda today announced that the U.S. FDA granted breakthrough therapy designation for its investigational drug pevonedistat for the treatment of patients with higher-risk myelodysplastic syndromes (HR-MDS). Pevonedistat, a first in class NEDD8-activating enzyme inhibitor, could be the first novel treatment for HR-MDS patients in more than a decade, expanding treatment options that have so far been limited to hypomethylating agent monotherapy alone. Even with current treatment options, outcomes for people living with HR-MDS remain poor.
The breakthrough therapy designation is based on the final analysis of the Pevonedistat-2001 Phase 2 study, which evaluated pevonedistat plus azacitidine versus azacitidine alone in patients with rare leukemias, including HR-MDS.
