Takeda’s Pevonedistat Gets Breakthrough Therapy Designation

The FDA granted a breakthrough therapy designation to Takeda Pharmaceuticals’ pevonedistat for patients with higher-risk myelodysplastic syndromes (HR-MDS), a rare form of bone marrow cancer.

The designation was granted based on a phase 2 clinical trial of pevonedistat plus azacitidine versus azacitidine alone in patients with rare leukemias, including HR-MDS.

Pevonedistat is currently in phase 3 studies as a first-line treatment for patients with HR-MDS and higher-risk chronic myelomonocytic leukemia.