Ultragenyx Pharmaceutical Inc.

Ultragenyx Enters into Agreement to Sell Rare Pediatric Disease Priority Review Voucher for $203 Million

Ultragenyx agreed to sell a Rare Pediatric Disease Priority Review Voucher for $203 million in non-dilutive funding. The PRV was earned from the FDA approval of FAYUVI (UX111), the first FDA-approved treatment for MPS IIIA (Sanfilippo syndrome Type A). Closing is subject to customary conditions including HSR antitrust clearance; management framed the proceeds as supporting the company's path to profitability.

•18d ago

Ultragenyx Enters into Agreement to Sell Rare Pediatric Disease Priority Review Voucher for $210 Million

Ultragenyx agreed to sell a Rare Pediatric Disease Priority Review Voucher for $210 million, generating significant non-dilutive capital. The PRV was awarded upon FDA approval of GENGLYCOS (DTX401), the first treatment targeting the underlying cause of glycogen storage disease type Ia. Management says proceeds advance rare-disease pipeline programs and support the path to profitability; closing remains subject to Hart-Scott-Rodino clearance.

•25d ago

Ultragenyx Announces Marketing Authorisation Application (MAA) Submission to the European Medicines Agency (EMA) for the First Investigational Gene Therapy for MPS IIIA (Sanfilippo Syndrome Type A)

Ultragenyx announced the EMA has validated its Marketing Authorisation Application for rebisufligene etisparvovec, an investigational AAV9 gene therapy for MPS IIIA (Sanfilippo syndrome Type A). Validation starts formal scientific review but is not an approval; the therapy carries PRIME and Orphan Drug designations in Europe and received FDA approval on September 17, 2026. The company is also engaging with the UK MHRA and Saudi SFDA as it pursues geographic expansion; MPS IIIA affects an estimated 3,000-5,000 patients in commercially accessible geographies with a median life expectancy of 15 years.

•1mo ago

Ultragenyx Announces Approval of FAYUVI™ Gene Therapy, the First-Ever FDA-Approved Treatment for Sanfilippo Syndrome Type A (MPS IIIA)

The FDA granted standard full approval of FAYUVI (rebisufligene etisparvovec-hopf), also known as UX111, making it the first-ever FDA-approved treatment for Sanfilippo syndrome Type A, a fatal pediatric neurodegenerative disease. It is Ultragenyx's second gene therapy approval and sixth FDA approval overall, and the company received a Priority Review Voucher. Commercial product is expected to ship to Qualified Treatment Centers within 30-60 days, supported by the UltraCare access program, with manufacturing entirely US-based at Ultragenyx's Bedford, Massachusetts facility and Andelyn Biosciences in Columbus, Ohio. Approval rests on the pivotal Transpher A trial, where FAYUVI-treated patients (mITT, N=17) showed a 23.5-point higher Bayley-III cognitive score versus an external natural history cohort (N=27), with p<0.0001; safety labeling includes hepatotoxicity, thrombocytopenia, and TMA monitoring. Sanfilippo syndrome Type A affects an estimated 3,000 to 5,000 patients in commercially accessible geographies; the asset was originally developed at Nationwide Children's Hospital and out-licensed to Ultragenyx by Abeona after funding constraints.

•1mo ago

Ultragenyx Announces the Publication of a Successful 96-Week Randomized, Placebo-Controlled Trial with Crossover Treatment of GENGLYCOS™ (also known as DTX401) AAV Gene Therapy in GSDIa in The Journal of Inherited Metabolic Disease

Ultragenyx announced the publication of 96-week data from the Phase 3 study of GENGLYCOS (DTX401) in The Journal of Inherited Metabolic Disease, showing a 61% mean reduction in daily cornstarch intake across both treatment and crossover groups. Participants maintained glycemic control and eliminated nighttime cornstarch dosing in 33% of the original treatment group and 42% of the crossover group, demonstrating durable efficacy of the gene therapy approved earlier this year. The therapy demonstrated an acceptable safety profile through week 96 with no AAV8 class effects observed, supporting the confirmatory trial requirements for its accelerated approval.

•2mo ago

Ultragenyx Announces U.S. FDA Approval of GENGLYCOS™ Gene Therapy, the First-Ever FDA-Approved Treatment Designed to Treat the Underlying Cause of Glycogen Storage Disease Type Ia (GSDIa)

Ultragenyx received FDA accelerated approval for GENGLYCOS, the first gene therapy to treat the underlying cause of glycogen storage disease type Ia (GSDIa) in patients eight years and older. The approval is based on positive Phase 3 data showing a statistically significant reduction in cornstarch requirements, a primary burden of the disease. The company also received a Priority Review Voucher and plans to launch the drug immediately through a network of Qualified Treatment Centers.

•2mo ago

Ultragenyx Reports Second Quarter 2026 Financial Results and Corporate Update

Ultragenyx reported record Q2 revenue of $214 million, a 28% increase from the prior year, driven by strong sales of Crysvita ($156M) and Dojolvi ($27M). The company narrowed its net loss to $92 million, or $0.90 per share, compared to a loss of $115 million in Q2 2025, and reaffirmed 2026 revenue guidance of $730 million to $760 million. Management reiterated its target to reach profitability in 2027 and highlighted upcoming catalysts, including PDUFA dates for DTX401 (Aug 23) and UX111 (Sept 19), and a pivotal data readout for GTX-102 in the fall.

•2mo ago

Calidi Biotherapeutics Appoints Former Genentech/Roche and Celgene Executive Corsee Sanders, Ph.D., to Board of Directors

Calidi Biotherapeutics appointed Corsee Sanders, Ph.D., a biopharma industry veteran with 23 years of experience at Genentech/Roche, as an independent director to its board. Dr. Sanders previously served as an executive at Juno and advised Celgene and Bristol Myers Squibb following acquisitions, bringing significant clinical development and integration expertise. She currently serves on the boards of Legend Biotech and Ultragenyx, and will support Calidi as it advances its CLD-401 candidate and RedTail platform.

•4mo ago

Ultragenyx Reports First Quarter 2026 Financial Results and Corporate Update

Ultragenyx reported Q1 2026 revenue of $136 million, slightly down from the prior year, but reaffirmed full-year 2026 revenue guidance of $730 million to $760 million and maintained its path to profitability in 2027. The pipeline delivered significant catalysts, including positive top-line Phase 3 data for DTX301 showing a statistically significant 18% reduction in plasma ammonia, while GTX-102 Phase 3 data are expected in the second half of 2026. Regulatory progress continues with the FDA assigning a PDUFA action date of August 23, 2026 for DTX401 and September 19, 2026 for UX111.

•5mo ago