Ultragenyx Shares Climb Following FDA Approval of Fayuvi

Previously known as UX111, the gene therapy targets a rare, inherited condition that inflicts severe damage on the brain and nervous system. Before this decision, medical interventions for the syndrome were restricted to symptom management, leaving families without a disease-modifying option.

Ultragenyx plans to begin shipping the product to qualified treatment centers within the next 30 to 60 days. This milestone represents the company’s second gene therapy approval and its sixth overall product clearance. Additionally, the firm received a priority review voucher as part of the regulatory approval process.

Despite the positive news, the company has faced significant market pressure throughout the year, with shares down 37% as of Thursday’s close. Investor sentiment was recently dampened earlier this month when the company reported unfavorable Phase 3 trial results for its Angelman syndrome candidate, apazunersen.