Ultragenyx获FDA加速批准GSDIa基因疗法GENGLYCOS

Ultragenyx Pharmaceutical Inc. (RARE) announced that the U.S. FDA has granted accelerated approval for GENGLYCOS, also known as DTX401, in adult and pediatric patients eight years and older with glycogen storage disease type Ia.
Glycogen storage disease type Ia (GSDIa), also known as Von Gierke disease is an ultra-rare genetic metabolic disorder caused by a deficiency of the enzyme needed to release glucose from the liver into the bloodstream. Patients face life-threatening hypoglycemia episodes and other complications, requiring a burdensome regimen of raw cornstarch intake around the clock as an oral glucose replacement therapy. The condition affects an estimated 1,500-2,500 patients in the U.S. and 6,000-8,000 worldwide.
GENGLYCOS is the first FDA-approved therapy designed to directly address the root cause of GSDIa. Clinical studies showed reduced reliance on cornstarch and improved ability to regulate glucose during fasting or metabolic stress, alleviating disease burden and lowering the risk of severe hypoglycemia. GENGLYCOS is composed of a recombinant, non-replicating AAV8 vector whose therapeutic design enables sustained glucose regulation.
The approval was based on the 48-week randomized, double-blind, placebo-controlled Phase 3 GlucoGene study which treated 46 participants aged eight years and older with DTX401 (1.0 x 10^13 GC/kg dose) or placebo. Results showed a significant reduction in cornstarch requirements in the treated group (p<0.001). At Week 48, eligible participants crossed over to receive the alternate treatment, with follow-up analyses conducted at Week 96 and Week 144 confirming durable benefit.
"This approval represents an important achievement for our company and the realization of the promise of a powerful new tool to deliver transformative medicines for people living with rare diseases." said Eric Crombez, M.D., chief medical officer at Ultragenyx.
David Weinstein, M.D., MMSc, a leading expert in GSDIa, noted that day-to-day management of the disease requires relentless adherence to cornstarch intake, with any lapse risking seizures or death. He called the approval " a major step forward for the GSDIa community" after nearly 30 years of scientific progress.
This marks Ultragenyx's first gene therapy approval and its fifth FDA approval overall. The company also received a Priority Review Voucher upon approval.
RARE has traded between $18.29 and $39.89 over the past year. The stock closed Wednesday's trading at $26.24, up 2.94%. In pre-market trading session, the stock is at $30.56, up 16.45%.
Competitor Landscape
Several other companies are pursuing early-stage programs in GSDIa.
Beam Therapeutics is advancing BEAM-301, a Phase 1/2 base-editing candidate targeting the common G6PC mutation. Moderna previously initiated mRNA-375, an mRNA-based therapy encoding G6Pase-a, through the program remains at proof-of-concept stage following pipeline reprioritization.
Additional academic and industry groups are exploring alternative AAV-based gene therapies and genome editing strategies, through these remain in preclinical development.
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