Ultragenyx Shares Soar 15% on Landmark FDA Filing for Gene Therapy
Company completes submission for DTX401, a potential first-in-class treatment for the underlying cause of the rare metabolic disorder GSDIa.
Shares of Ultragenyx Pharmaceutical (RARE) surged more than 15% in Tuesday trading after the company announced it had completed its rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration for its gene therapy, DTX401.
The therapy is aimed at treating Glycogen Storage Disease Type Ia (GSDIa), a rare and life-threatening genetic disorder. The stock climbed $3.06 to close at $22.78, marking a significant vote of investor confidence in the therapy's potential. If approved, DTX401 would be the first-ever medical therapy to treat the underlying cause of the disease.
Glycogen Storage Disease Type Ia, also known as von Gierke disease, is an inherited metabolic disorder caused by a faulty gene. According to the U.S. National Library of Medicine, this defect impairs the liver's ability to break down stored glycogen into glucose, the body's main source of energy. This can lead to severe and dangerous low blood sugar levels (hypoglycemia), organ damage, and a host of other metabolic complications. Currently, there are no approved pharmacological treatments; management relies on a burdensome regimen of continuous glucose and cornstarch feedings, day and night, to prevent life-threatening hypoglycemia.
Ultragenyx's DTX401 is an investigational adeno-associated virus (AAV) gene therapy designed to deliver a stable, functional copy of the corrective gene, potentially allowing the body to properly regulate blood sugar. According to the company's official announcement, the BLA submission is supported by data from 52 patients with up to six years of follow-up. The Phase 3 study showed that treatment with DTX401 led to a significant reduction in the need for daily cornstarch, a key measure of the treatment's efficacy, while maintaining stable glucose levels.
"The completion of our BLA submission for DTX401 is a critical milestone for the GSDIa community and brings us one step closer to potentially providing the first-ever therapy that addresses the underlying disease," the company stated in its press release.
The regulatory pathway for DTX401 has been bolstered by several special designations from the FDA, including Orphan Drug, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT). These designations are intended to facilitate the development and expedite the review of drugs that treat serious conditions and fill an unmet medical need.
Despite Tuesday's sharp rally, Ultragenyx's stock remains well below its 52-week high of $46.50. However, the analyst community remains broadly positive on the company's prospects. The stock carries a strong consensus "Buy" rating from over 20 analysts, with an average 12-month price target of approximately $81. This suggests Wall Street sees significant long-term value, contingent on pipeline execution and regulatory successes like the one announced today.
With a market capitalization of approximately $3.3 billion, Ultragenyx has established itself as a key player in the development of therapies for rare and ultra-rare diseases. The completion of the BLA for DTX401 is a pivotal step for the company as it seeks to transition from a clinical-stage to a commercial-stage powerhouse in the gene therapy space. The FDA will now review the complete application and decide whether to accept it for a full review, a process that typically takes 60 days.