Also known as: Ultragenyx, Ultragenyx Pharmaceutical
Ultragenyx Pharmaceutical Inc. is an American biopharmaceutical company focused on developing therapies for rare and ultra-rare genetic diseases. Founded in 2010 and headquartered in Novato, California, it trades on Nasdaq under RARE.
Ultragenyx began a restructuring plan that includes a 10% workforce reduction and termination of UX143 manufacturing agreements. The company also faces a class action tied to setrusumab disclosures.
Class action lawsuits allege false statements about clinical trial results and regulatory prospects at Atara Biotherapeutics, Ultragenyx, Mereo BioPharma, and Vistagen Therapeutics. The complaints cite failed Phase 3 studies for setrusumab in Osteogenesis Imperfecta and fasedienol for social anxiety disorder, along with manufacturing issues affecting Atara's tabelecleucel application. Investors have deadlines ranging from April to May 2026 to seek lead plaintiff status.
Opus Genetics reported positive early data from its BEST1 gene therapy program and expects FDA action on its presbyopia treatment in October 2026. Ultragenyx announced 2025 revenue of $673 million and initiated a strategic restructuring plan targeting profitability in 2027.
The FDA has accepted Ultragenyx's BLA for DTX401, an AAV gene therapy for Glycogen Storage Disease Type Ia, granting Priority Review with a PDUFA action date of August 23, 2026.
Ultragenyx Pharmaceutical confronts multiple shareholder class action lawsuits over Phase III trial disclosures while reporting positive UX111 gene therapy data and implementing a 10% workforce reduction.
The global antibodies market is projected to reach $667.8 billion by 2032 with a 12.9% CAGR, while high throughput antibody production services are expected to grow at 11.2% CAGR through 2033, driven by biopharmaceutical development and monoclonal antibody therapeutics demand.
Ultragenyx Pharmaceutical announced positive long-term clinical data for UX111 gene therapy for Sanfilippo syndrome Type A, with up to 8.5 years of follow-up showing sustained biomarker reductions and functional improvements. The company resubmitted its BLA to the FDA in January 2026.
Ultragenyx Pharmaceutical received an Incomplete Response Letter from the FDA for its UX111 gene therapy resubmission for Sanfilippo syndrome type A, requesting additional documentation. The company announced a 10% workforce reduction and expects profitability by 2027.
Ultragenyx will lay off 130 employees as part of restructuring aimed at profitability by 2027. The FDA rejected the company's gene therapy application for Sanfilippo syndrome due to manufacturing concerns.
Ultragenyx reported Q4 2025 revenues of $207 million, up 25% year-over-year, while announcing a 10% workforce reduction and resubmission of its UX111 gene therapy application following positive long-term clinical data.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07511556 |
First-in-human Study of UX016 in GNEM |
NOT_YET_RECRUITING | PHASE1/PHASE2 |
| NCT07447648 |
Assessing the Impact of Intensification of Lipid Lowering Therapy With Guidelines-based Evinacumab Administration on Coronary Plaque Volumes Measured by Coronary Computed Tomography Angiography (CCTA) in Patients With Homozygous Familial Hypercholesterolemia (HoFH) |
RECRUITING | |
| NCT07159581 |
Gene Therapy for Wilson Disease Evaluated by 64Cu PET/CT |
ENROLLING_BY_INVITATION | |
| NCT07157254 |
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS) |
RECRUITING | PHASE2 |
| NCT07097311 |
Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) |
RECRUITING | PHASE2 |
| NCT06636383 |
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program |
RECRUITING | |
| NCT06636071 |
Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT06617429 |
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT06415344 |
Long-term Extension of GTX-102 in Angelman Syndrome |
ENROLLING_BY_INVITATION | PHASE3 |
| NCT06340685 |
Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency |
RECRUITING | PHASE1 |
