If at first you don’t succeed in gene therapy, then try, try again. That's the lesson Ultragenyx likely took home this week as it pocketed its first FDA nod in the class just over a year after a separate program stalled on manufacturing grounds.
Late Wednesday, the FDA gave an accelerated go-ahead to pariglasgene brecaparvovec-opnr, Ultragenyx’s AAV gene therapy for the rare genetic disorder glycogen storage disease type Ia (GSDIa), which will now head to market with the brand name Genglycos.
The accelerated approval specifically covers the gene therapy—previously coded DTX401—in adults and kids over the age of 8 years with GSDIa, an ultrarare genetic disorder driven by a deficiency of the enzyme G6PC needed to release glucose from the liver to the bloodstream. The liver is less capable of controlling glucose levels in these patients, leading to potentially life-threatening hypoglycemia episodes and other complications.
By Ultragenyx’s reckoning, there are between 1,500 and 2,500 people living with GSDIa in the U.S. out of an estimated global patient population of 6,000 to 8,000 “within commercially accessible geographies,” the company said in an Aug. 19 release.
The green light marks Ultragenyx’s fifth from the FDA and its first for a gene therapy. The company has also secured a valuable priority review voucher in tandem with its approval.
