Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) shares are up during Thursday’s premarket session on the recent U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy, Genglycos (pariglasgene brecaparvovec-opnr, or DTX401).
Genglycos Approved For Ultra-Rare Genetic Disorder
This marks a significant milestone as it is the first-ever FDA-approved treatment for Glycogen Storage Disease Type Ia (GSDIa).
The approval of Genglycos represents Ultragenyx’s first gene therapy and fifth FDA approval.
GSDIa is an ultra-rare genetic metabolic disorder caused by a deficiency of the enzyme needed to release glucose from the liver to the bloodstream.
The deficiency reduces the liver’s ability to control glucose levels and is associated with potentially life-threatening hypoglycemia episodes and other serious complications.
GSDIa affects 1,500-2,500 patients in the U.S. and 6,000-8,000 worldwide.
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 eight years and older with DTX401 (1.0 x 10^13 GC/kg dose) or placebo, showing a reduction in the cornstarch requirements in the treated group.
As part of accelerated approval, Ultragenyx has agreed to provide two years of safety and efficacy clinical data from open-label commercial treatment of 50 patients and 20 control patients through enhancement of its existing GSDIa Disease Monitoring Program (DMP).
The DMP will also evaluate previously treated clinical trial participants as well as these new commercial patients for a total of 10 years.
Analyst Bullish
Citing a conference call, William Blair noted the wholesale acquisition cost for Genglycos will be $2.7 million per patient.
Analyst Sami Corwin said, “We are encouraged by the approval of Genglycos, which we believe is incrementally positive to Ultragenyx’s stock.”
Analyst Corwin resumed coverage of Ultragenyx with an Outperform rating and currently models $362 million in peak sales for Genglycos.
RARE Stock Price Activity: Ultragenyx Pharmaceutical shares were up 11.28% at $29.20 during premarket trading on Thursday, according to Benzinga Pro data.
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