Ultragenyx Pharmaceutical
Ultragenyx Pharmaceutical works on 18 rare diseases tracked on Trial Friend, including Amyotrophic Lateral Sclerosis, Angelman Syndrome, Barth Syndrome and 15 more, with 12 active clinical trials (2 recruiting) and 3 FDA-approved rare disease drugs.
Ultragenyx Pharmaceutical is a biopharmaceutical company dedicated to treating rare and ultra-rare genetic diseases. The company's portfolio includes 6 approved therapies: Crysvita (burosumab) for X-linked hypophosphatemia, Mepsevii (vestronidase alfa) for MPS VII, Dojolvi (triheptanoin) for long-chain fatty acid oxidation disorders, Evkeeza (evinacumab), which Regeneron markets in the US and Ultragenyx sells outside the US under license, for homozygous familial hypercholesterolemia, Genglycos (pariglasgene brecaparvovec-opnr), which received FDA accelerated approval in August 2026 for glycogen storage disease type Ia, and Fayuvi (rebisufligene etisparvovec-hopf), formerly UX111, which received full FDA approval on September 17, 2026 as the first-ever treatment for Sanfilippo syndrome type A (MPS IIIA). Genglycos and Fayuvi are the company's first 2 gene therapies to reach the market. Its pipeline includes DTX301 gene therapy for ornithine transcarbamylase (OTC) deficiency, a urea cycle disorder, which met the first of its 2 main Phase 3 goals in March 2026, and UX701 gene therapy for Wilson disease, now in the dose-finding stage of a combined Phase 1/2/3 trial. In 2 other programs the Phase 3 trials missed their main goals, setrusumab for osteogenesis imperfecta in December 2025 and apazunersen (GTX-102) for Angelman syndrome on September 2, 2026, and Ultragenyx says it is reviewing next steps for both.
Ultragenyx Pharmaceutical Drug Pipeline
Ultragenyx Pharmaceutical has 12 active clinical trials across 3 development stages, with 2 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.
Ultragenyx Pharmaceutical Clinical Trials (12)
Active and recruiting clinical trials sponsored by Ultragenyx Pharmaceutical, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
Ultragenyx Pharmaceutical FDA-Approved Drugs (3)
Medications developed or marketed by Ultragenyx Pharmaceutical that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.
| Drug Name | Brand Name | Rare Diseases | Approval Date |
|---|---|---|---|
| TRIHEPTANOIN Medium-chain Triglyceride [EPC] | DOJOLVI oral | — | Jun 30, 2020 |
| VESTRONIDASE ALFA Lysosomal beta Glucuronidase [EPC] | MEPSEVII intravenous | — | Nov 15, 2017 |
| REBISUFLIGENE ETISPARVOVEC | FAYUVI | — |
Ultragenyx Pharmaceutical Trial Locations
Ultragenyx Pharmaceutical clinical trials are running at 235 sites in 18 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (18)
Diseases targeted by Ultragenyx Pharmaceutical's clinical trial and drug development programs
Amyotrophic lateral sclerosis is a progressive neurodegenerative disease destroying motor neurons in the brain and spinal cord. This causes progressive weakness and paralysis of voluntary muscles whil...
Angelman Syndrome is a rare neurological disorder caused by loss of function of the UBE3A gene on the maternal chromosome 15. People with this condition typically develop normal until 6-12 months of a...
Barth syndrome is an ultra-rare X-linked genetic disorder caused by mutations in the TAFAZZIN gene, which produces an enzyme essential for remodeling cardiolipin, a phospholipid that mitochondria need...
Glycogen Storage Disease Type II (GSD II, also called Pompe Disease) is a lysosomal storage disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA), which breaks down glycogen. This l...
Hemophilia B is an X-linked bleeding disorder caused by deficiency or dysfunction of clotting factor IX. Clinically indistinguishable from Hemophilia A, the key difference is factor type. Modern facto...
Homozygous Familial Hypercholesterolemia is a severe genetic disorder causing extremely high cholesterol levels from birth, leading to early heart disease and cholesterol deposits in tendons and aroun...
Patient Resources
Organizations and resources related to Ultragenyx Pharmaceutical's rare disease focus areas
Frequently Asked Questions About Ultragenyx Pharmaceutical
Common questions about Ultragenyx Pharmaceutical's rare disease programs, clinical trials, and treatments.