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First Therapy Approved for Dangerous Metabolic Disorder
Approval was based on a 48-week trial in 46 participants that showed a significant reduction in cornstarch requirements, Ultragenyx said.
- On Wednesday, the FDA granted accelerated approval to Ultragenyx Pharmaceutical Inc.'s GENGLYCOS for patients eight years and older with glycogen storage disease type Ia, marking the company's first-ever gene therapy approval.
- Patients with GSDIa, an ultra-rare metabolic disorder caused by G6Pase enzyme deficiency, require a relentless, round-the-clock regimen of raw cornstarch to manage blood sugar and avoid potentially life-threatening hypoglycemia episodes.
- Data from the 48-week Phase 3 GlucoGene trial in 46 participants showed a statistically significant reduction in cornstarch requirements , though ALT/AST enzyme elevations occurred in 71% of treated patients.
- Ultragenyx set a U.S. list price of $2.7 million per patient for the therapy, which will be available through a national network of Qualified Treatment Centers within 30 to 60 days.
- Accelerated approval requires confirmatory clinical benefit data, with Ultragenyx agreeing to monitor 50 patients and 20 control patients; Analyst Sami Corwin models $362 million in peak sales for the therapy.
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First Therapy Approved for Dangerous Metabolic Disorder
(MedPage Today) -- The FDA on Wednesday granted accelerated approval to pariglasgene brecaparvovec (Genglycos) as the first treatment for glycogen storage disease type Ia (GSDIa), an inherited metabolic disorder that requires frequent consumption...
·New York, United States
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