Ultragenyx Pharmaceuticals Inc. surged 11.1% in pre-market trading on Thursday after U.S. regulators approved the company’s gene therapy for a rare metabolic disorder.
The stock was last quoted at $29.16, near its highest level in several weeks, while the broader market benchmarks showed little movement. The S&P 500 was essentially flat, the Dow Jones Industrial Average slipped modestly, and the Nasdaq Composite was nearly unchanged.
The U.S. Food and Drug Administration granted accelerated approval for GENGLYCOS (pariglasgene brecaparvovec-opnr), also designated DTX401, for the treatment of glycogen storage disease type Ia (GSDIa) in patients aged eight and older. The approval came with a Priority Review Voucher, a regulatory incentive designed to accelerate the development of therapies for rare conditions.
GSDIa is a genetic disorder affecting an estimated 1,500 to 2,500 patients in the United States. GENGLYCOS is the first therapy to address the root cause of the disease rather than merely manage symptoms, according to the company. The approval is Ultragenyx’s fifth from the FDA and its first gene therapy authorization.
The decision was based on data from the 48-week Phase 3 GlucoGene study, which showed a statistically significant reduction in patients’ daily cornstarch requirements. Chief Medical Officer Eric Crombez stated that the therapy’s ability to regulate glucose "has the potential to reduce the risk of severe or life-threatening hypoglycemia for patients."
Analysts at Citi maintained a Buy rating and raised their price target to $58 from $45. H.C. Wainwright also reiterated its Buy rating with a $50 target. The company’s shares have gained roughly 20% over the past month, though they remain well below the 52-week high of $39.89.
