Ultragenyx won FDA accelerated approval for GENGLYCOS, its first gene therapy, for glycogen storage disease type Ia in patients aged eight and older. Shares jumped 14 percent in after-hours trading to $28.00. The approval is the company's fifth from the FDA and its first gene therapy.
Ultragenyx Pharmaceutical won FDA accelerated approval for GENGLYCOS, its first gene therapy, for glycogen storage disease type Ia in patients aged eight and older.
"The approval of GENGLYCOS fulfills our commitment to provide the first therapy that directly targets the root cause of GSDIa," Eric Crombez, chief medical officer at Ultragenyx, said.
The approval is based on the 48-week Phase 3 GlucoGene study of 46 participants, which showed a reduction in cornstarch requirements in the treated group compared with placebo (p<0.001, meaning less than 0.1 percent chance the result is random). GSDIa, an ultra-rare genetic metabolic disorder, affects 1,500-2,500 patients in the U.S. and 6,000-8,000 worldwide within commercially accessible geographies. Ultragenyx received a Priority Review Voucher upon approval.
Shares jumped 14 percent in after-hours trading to $28.00, after closing up 2.98 percent at $26.25. The approval is Ultragenyx's fifth from the FDA and its first gene therapy. As part of the accelerated approval, the company agreed to provide two years of safety and efficacy data from open-label commercial treatment of 50 patients and 20 controls through its GSDIa Disease Monitoring Program.
GENGLYCOS (pariglasgene brecaparvovec-opnr), also known as DTX401, delivers a working copy of the G6PC gene to restore the enzyme needed to release glucose from the liver. Patients currently manage the disease with a demanding around-the-clock regimen of raw cornstarch to avoid life-threatening hypoglycemia. The therapy will be available through a national network of Qualified Treatment Centers and is manufactured at Ultragenyx's Gene Therapy Manufacturing Facility in Bedford, Massachusetts.
The most common adverse reactions in the primary efficacy analysis period were elevated ALT/AST enzymes (71 percent), nausea (38 percent), hypertriglyceridemia (29 percent), adrenal insufficiency (24 percent), headache (24 percent), constipation (19 percent), acne (19 percent), hyperglycemia (14 percent), Cushingoid features (14 percent) and anaphylaxis (10 percent). Seven serious adverse events were observed, including anaphylaxis, adrenal insufficiency, high lactate and hypoglycemia. The label carries warnings for hypersensitivity reactions, immune-mediated hepatotoxicity, adrenal insufficiency and a theoretical risk of tumorigenicity from AAV vector integration.
The accelerated approval requires confirmatory data to verify clinical benefit, and continued approval may hinge on those results. Ultragenyx will host a conference call at 6 p.m. Eastern Time to discuss the approval, and investors will watch the post-marketing Disease Monitoring Program data as the next catalyst.
This article is for informational purposes only and does not constitute investment advice.
