Ultragenyx Pharmaceutical Inc. RARE
Ultragenyx Pharmaceutical Inc. (Nasdaq: RARE) is a rare genetic drug developer. Its lead program, Dojolvi (triheptanoin); portfolio also Crysvita, Mepsevii, is in Phase 3 for long-chain fatty acid oxidation disorders (LC-FAOD). Its next catalyst on record is GTX-102: Complete enrollment in Phase 2/3 Aurora study (H2 2026).
Upcoming catalysts
No catalyst is dated to the next 30 days or to an FDA action date.
Next further out: GTX-102 — Complete enrollment in Phase 2/3 Aurora study (H2 2026). 4 catalysts on record beyond the next 30 days; subscribers see each with its filing sentence.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| Dojolvi | LC-FAOD | small molecule | Approved |
| Mepsevii | MPS VII (Sly syndrome) | protein/peptide | Approved |
| Crysvita (with Kyowa Kirin) | X-linked hypophosphatemia and tumor-induced osteomalacia | antibody | Approved |
| DTX401 (pariglasgene brecaparvovec) | Glycogen storage disease type Ia | gene therapy | Phase 3 |
| UX111 (rebisufligene etisparvovec) | Sanfilippo syndrome type A (MPS IIIA) | gene therapy | Phase 3 |
| GTX-102 (apazunersen) | Angelman syndrome | RNA (siRNA/ASO/mRNA) | Phase 3 |
| DTX301 (avalotcagene ontaparvovec) | OTC deficiency | gene therapy | Phase 3 |
| UX143 (setrusumab) | Osteogenesis imperfecta | antibody | Phase 3 |
| UX701 (rivunatpagene miziparvovec) | Wilson disease | gene therapy | Phase 2 |
| UX016 | GNE myopathy | small molecule | Pre-clinical |
Recent results and decisions
- Aug 4, 2026approval Japan (ex-US): Dojolvi received conditional manufacturing and marketing approval on March 23, 2026 and was launched in Japan in May 2026 after NHI price listing.
In May 2026, Dojolvi was listed on the National Health Insurance (NHI) drug price list and was launched in Japan following the receipt of manufacturing and marketing approval under the Conditional Approval System for Pharmaceuticals on March 23, 2026.
Filing - May 5, 2026positive Longer-term Phase 1/2 data (74 patients treated, 66 in LTE) show durable, improving effects across multiple domains with a consistent safety profile; no formal endpoint stated.
Patients have continued to show positive improvements across multiple domains and continued to gain ground developmentally.
Filing - Apr 2, 2026accepted FDA accepted UX111 BLA resubmission for accelerated approval; PDUFA action date September 19, 2026.
The FDA set a Prescription Drug User Fee Act (PDUFA) action date of September 19, 2026.
Filing
4 more on record.
For subscribers
- Cash runway against each catalyst, with the company's own runway statement
- Track record: every Phase 3 readout, FDA decision and guidance slip since 2014
- Expected move for each dated catalyst, from comparable past events
- Phase 2 evidence behind each Phase 3 readout
- Companies on the same target or class
- Full catalyst list with filing sentences, deals, registered trials, Excel export
Other phase 3 rare genetic companies
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.