FDAnews Drug Daily Bulletin
Pharmaceuticals / Postmarket Safety / Research and Development

Zafgen to Continue Prader-Willi Syndrome Trial Despite Patient Death

Oct. 29, 2015

Boston drugmaker Zafgen plans to proceed with a Phase 3 trial of beloranib for the rare genetic metabolic disorder Prader-Willi syndrome, despite a patient death.

Results from that trial and a Phase 2b study evaluating the drug’s use in diabetes-linked obesity are expected by early 2016 and will be reviewed by the FDA to inform the design of a second Phase 3 trial, the company said last week. The randomized arms of both studies will be closed early to keep data integrity intact.

Last week, the FDA placed a partial hold on the Phase 3 trial after news of the patient death. The cause of death was respiratory failure from pulmonary emboli, and Zafgen says the connection between the death and beloranib remains unknown. The drug is a MetAP2 inhibitor.