Ultragenyx Pharmaceutical Inc. RARE
Ultragenyx Pharmaceutical Inc. (Nasdaq: RARE) is a rare genetic drug developer. Its lead program, DTX301, is in Phase 3 for Ornithine transcarbamylase (OTC) deficiency. Its next catalyst on record is GENGLYCOS and FAYUVI PRV sales: FAYUVI PRV sale closure (Q4 2026).
Upcoming catalysts
No catalyst is dated to the next 30 days or to an FDA action date.
Next further out: GENGLYCOS and FAYUVI PRV sales — FAYUVI PRV sale closure (Q4 2026). 3 catalysts on record beyond the next 30 days; subscribers see each with its filing sentence.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| CRYSVITA | X-Linked Hypophosphatemia (XLH) and Tumor-Induced Osteomalacia (TIO) | antibody | Approved |
| MEPSEVII | Mucopolysaccharidosis VII (MPS VII) | protein/peptide | Approved |
| DOJOLVI | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | small molecule | Approved |
| EVKEEZA | Homozygous Familial Hypercholesterolemia (HoFH) | antibody | Approved |
| GENGLYCOS (DTX401) | Glycogen Storage Disease Type Ia (GSDIa) | gene therapy | Approved |
| FAYUVI | Mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome Type A) | gene therapy | Approved |
| DTX301 | Ornithine transcarbamylase (OTC) deficiency | gene therapy | Phase 3 |
| UX143 (setrusumab) | Osteogenesis Imperfecta (OI) | antibody | Phase 3 |
| GTX-102 (apazunersen) | Angelman syndrome | RNA (siRNA/ASO/mRNA) | Phase 3 |
| UX701 | Wilson disease | gene therapy | Phase 2 |
| UX016 | GNE myopathy (GNEM) | small molecule | Phase 1 |
| UX111 | Not specified | gene therapy | Pre-clinical |
Recent results and decisions
- Nov 3, 2026negative UX701 demonstrated clinical activity in some patients but did not meet criteria to advance to Phase 3; development terminated.
Additional data in the dose-finding stage of the Cyprus2+ study of UX701 for the treatment of Wilson disease, including data from the fourth dosing cohort, demonstrated clinical activity in some patients, but did not meet the criteria that the majority of patients achieve biochemical response and el
Filing - approval FDA granted standard full approval for FAYUVI as a treatment of pediatric patients with Sanfilippo syndrome Type A.
In September 2026, the FDA granted standard full approval for FAYUVI as a treatment of pediatric patients with Sanfilippo syndrome Type A.
Filing - Aug 19, 2026approval FDA granted accelerated approval for GENGLYCOS as a treatment for adult and pediatric patients eight years and older with GSDIa.
In August 2026, the U.S. Food and Drug Administration (FDA) granted accelerated approval for GENGLYCOS as a treatment for adult and pediatric patients eight years and older with GSDIa.
Filing
6 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.