Ultragenyx Wins FDA Approval for First Gene Therapy Genglycos

Ultragenyx Pharmaceutical Inc. shares rose in Thursday's premarket trading after the U.S. Food and Drug Administration recently granted accelerated approval for its gene therapy Genglycos.

Genglycos Approved for Ultra-Rare Genetic Disease

This marks a significant milestone as it is the first FDA-approved treatment for glycogen storage disease type I.

The approval of Genglycos represents Ultragenyx's first gene therapy and its fifth FDA approval.

GSDIa is an ultra-rare inherited metabolic disease caused by a defect in the enzyme required for the liver to release glucose into the blood.

This defect reduces the liver's ability to control blood sugar levels and is associated with potentially life-threatening hypoglycemic episodes and other serious complications.

GSDIa affects 1,500-2,500 patients in the U.S. and 6,000-8,000 patients globally.

Phase 3 Data Supports FDA Approval

The approval of Genglycos is based on data from the 48-week Phase 3 GlucoGene study, which treated 46 participants aged 8 and older with DTX401 or placebo, showing a reduction in cornstarch requirements in the treatment group.

As part of the accelerated approval, Ultragenyx agreed to provide two-year safety and efficacy clinical data from open-label commercial treatment of 50 patients and 20 control patients by strengthening its existing GSDIa disease monitoring program.

The DMP will also evaluate participants previously treated in clinical trials as well as these new commercial patients for a total of 10 years.

Analysts Bullish

William Blair, citing a conference call, noted that Genglycos has a wholesale acquisition cost of $2.7 million per patient.

Analyst Sami Corwin stated, "We are encouraged by the approval of Genglycos, and we view this as incrementally positive for Ultragenyx shares."

Analyst Corwin resumed coverage of Ultragenyx with an Outperform rating and now forecasts peak sales of $362 million for Genglycos.

RARE Stock Activity: In Thursday's premarket trading, Ultragenyx Pharmaceutical shares rose 11.28% to $29.20.