Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) was up 7.5% in after-hours trading as of approximately 5:33 p.m. ET Wednesday after the FDA approved Genglycos for glycogen storage disease type Ia, or GSDIa. The one-time gene therapy is the first approved treatment designed to address the disorder's underlying cause. It will carry a U.S. list price of approximately $2.7 million per patient and is expected to become available through qualified treatment centers within 30 to 60 days.
The approval gives Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) first-mover status in a disease the company estimates affects only 1,500 to 2,500 Americans. The commercial question is whether a high-priced treatment for an ultra-rare population can produce meaningful revenue after diagnosis, reimbursement, and treatment-center constraints.
GSDIa prevents the liver from releasing glucose properly, leaving patients dependent on frequent raw-cornstarch doses to avoid potentially life-threatening hypoglycemia. In the Phase 3 GlucoGene trial, Genglycos reduced mean daily cornstarch intake by 41% at Week 48, compared with 10% for placebo, while maintaining glucose control. For Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), reducing that daily burden creates a clear argument for premium pricing.
Longer-term data also support the treatment's potential durability. At Week 96, the original-treatment group reported a 61% mean reduction from baseline, while the crossover group reported a 61% reduction from Week 48, when it began treatment. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) will manufacture the therapy at its Bedford, Massachusetts, facility, giving the company direct control over an important part of the supply chain.
The launch will also test infrastructure that could support future gene therapies. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) already sells rare-disease products including Crysvita, Dojolvi, Evkeeza and Mepsevii. Second-quarter revenue reached $214 million, while cash, cash equivalents, and marketable securities totaled $436 million as of June 30. Genglycos therefore enters a functioning commercial organization rather than a first-time launch platform.
#treatment
The approval gives Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) first-mover status in a disease the company estimates affects only 1,500 to 2,500 Americans. The commercial question is whether a high-priced treatment for an ultra-rare population can produce meaningful revenue after diagnosis, reimbursement, and treatment-center constraints.
GSDIa prevents the liver from releasing glucose properly, leaving patients dependent on frequent raw-cornstarch doses to avoid potentially life-threatening hypoglycemia. In the Phase 3 GlucoGene trial, Genglycos reduced mean daily cornstarch intake by 41% at Week 48, compared with 10% for placebo, while maintaining glucose control. For Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), reducing that daily burden creates a clear argument for premium pricing.
Longer-term data also support the treatment's potential durability. At Week 96, the original-treatment group reported a 61% mean reduction from baseline, while the crossover group reported a 61% reduction from Week 48, when it began treatment. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) will manufacture the therapy at its Bedford, Massachusetts, facility, giving the company direct control over an important part of the supply chain.
The launch will also test infrastructure that could support future gene therapies. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) already sells rare-disease products including Crysvita, Dojolvi, Evkeeza and Mepsevii. Second-quarter revenue reached $214 million, while cash, cash equivalents, and marketable securities totaled $436 million as of June 30. Genglycos therefore enters a functioning commercial organization rather than a first-time launch platform.
#treatment
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