FDA’s Marty Makary defends rare disease drug rejections, Vinay Prasad

WASHINGTON — Food and Drug Administration Commissioner Marty Makary defended the agency’s recent rejections of rare disease drugs in an interview with CNBC on Thursday. He also defended top FDA official Vinay Prasad, who oversees the center that rejected many of those drugs. 

In his defense, Makary appeared to reference the FDA’s stance on a gene therapy made by UniQure to treat Huntington’s disease, a rare neurodegenerative condition. The company is still in talks with the agency on whether its data are sufficient for review, and has said the FDA suddenly changed course on whether its clinical trial design is adequate. 

“There was a product where the researchers drilled a burr hole, literally a hole in people’s skulls, to inject intrathecally into the ventricle,” Makary said. “At the end of the randomization period, it found no benefit, and yet, this is one of the drugs that we were pressured to approve.”