RARE
Ultragenyx Pharmaceutical Inc.
September 17, 2026 · intraday
- Rarity
- 1.77
- Peak move
- +12.64%
- Float cap
- $1.2B
Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company that develops and commercializes therapies for patients with serious rare and ultra-rare genetic diseases. Its approved products treat X-linked hypophosphatemia, mucopolysaccharidosis VII, long-chain fatty acid oxidation disorders, and homozygous familial hypercholesterolemia. It commercializes through its own patient-focused organization in North America, Europe, Latin America, Asia, and select international markets, supplemented by third-party distributors.
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What we found
4 events · 2 from SEC or newswire · 1 timed with the move- Timed with the move
regulatory_approvalThe U.S. FDA approved Fayuvi (rebisufligene etisparvovec-hopf, UX111), Ultragenyx's AAV9 gene therapy, for pediatric patients with Sanfilippo syndrome type A (mucopolysaccharidosis type IIIA), the first approved treatment for the condition, and Ultragenyx received a Priority Review Voucher; the approval was announced on September 17, 2026, and the FDA had declined the therapy in 2025 over manufacturing concerns.
regulatory_approvalUltragenyx's UX111 (rebisufligene etisparvovec) AAV9 gene therapy for Sanfilippo syndrome type A faces an FDA PDUFA target action date of September 19, 2026, following a July 2025 Complete Response Letter citing CMC and facility-inspection issues.
class_action_filedPomerantz LLP announced it is investigating claims on behalf of investors of Ultragenyx Pharmaceutical Inc.
insider_saleUltragenyx SVP and Chief Accounting Officer Theodore Alan Huizenga sold 1,283 shares on September 15, 2026 for about $17,410 to $17,462 at $13.57 to $13.61 per share under a 10b5-1 plan, leaving him 67,863 shares directly held, per SEC filings; he had also sold 161 shares on September 3, 2026.