Alnylam Wins FDA OK for First Therapy for Rare Blood Disorder

The first treatment for adult patients who suffer from a rare inherited blood disorder that increases the production of toxic molecules passed the FDA, the agency said Nov. 20.

Alnylam Pharmaceuticals Inc.'s drug, Givlaari, is intended to treat acute hepatic porphyria, which can result in a series of neurological attacks such as seizures, paralysis, respiratory failure, severe abdominal and back pain, and mental status changes, according to the Food and Drug Administration.

“These attacks occur suddenly and can produce permanent neurological damage and death,” Richard Pazdur, the director of the agency’s Oncology Center of Excellence and acting director of the ...