FDA approves first treatment for rare genetic enzyme disorder

The US Food and Drug Administration yesterday approved Mepsevii (vestronidase alfa-vjbk) to treat pediatric and adult patients with an inherited metabolic condition called mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome.

The drug was developed by US biotech firm Ultragenyx Pharmaceutical (Nasdaq: RARE), whose shares moved up 3.9% to $47.32 by close of trading on Wednesday. Ultragenyx recently gazumped Regenxbio to acquire all outstanding common stock of fellow Nasdaq-listed Dimension Therapeutics for about $151 million in cash.

“This approval underscores the agency’s commitment to making treatments available to patients with rare diseases,” said Julie Beitz, director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research (CDER). “Prior to today’s approval, patients with this rare, inherited condition had no approved treatment options,” she noted.