Ultragenyx Pharmaceutical

RARE Rare disease Lead: Approved ★ Read the deep dive → 12 Biotech Distilled articles ↓

Most recent annual filing: 2026-02-15

Catalyst timeline

UX016 Ph1 start2H 2026UX016 Ph1 start, 2H 2026, routine
UX701 Ph1 readout4Q 2026UX701 Ph1 readout, 4Q 2026, notable
DTX301 enrollment done1H 2027DTX301 enrollment done, 1H 2027, routine

Bar width shows how precise the guidance is — a pin is a stated date, a wide band a six-month window. Deeper blue means more of the thesis rides on the event; it is not a view on the stock.

Pipeline (12 active programs)

Crysvita

burosumab

Protein Target: FGF23

X-Linked Hypophosphatemia Approved subcutaneous Kyowa Kirin
Tumor-Induced Osteomalacia Approved subcutaneous Kyowa Kirin

Mepsevii

vestronidase alfa

Protein Target: beta-glucuronidase

Mucopolysaccharidosis VII Approved intravenous NCT03775174

Dojolvi

triheptanoin

Small Molecule

Long-Chain Fatty Acid Oxidation Disorders Approved oral NCT00947960

Evkeeza

evinacumab

Protein Target: ANGPTL3

Homozygous Familial Hypercholesterolemia Approved intravenous Regeneron NCT05611528

FAYUVI

rebisufligene etisparvovec · rebisufligene etisparvovec-hopf · UX111

Gene Therapy Target: SGSH

Sanfilippo Syndrome Type A Approved intravenous Abeona NCT02716246

GENGLYCOS

pariglasgene brecaparvovec · pariglasgene brecaparvovec-opnr · DTX401

Gene Therapy Target: G6Pase-α, G6PC, G6Pase

Glycogen Storage Disease Type Ia Approved intravenous NCT03517085

apazunersen

GTX-102

Nucleic Acid Target: UBE3A antisense, UBE3A-AS

Angelman Syndrome Phase 3 intrathecal NCT04259281

avalotcagene ontaparvovec

DTX301

Gene Therapy Target: OTC gene

Ornithine Transcarbamylase Deficiency Phase 3 intravenous NCT05345171

rivunatpagene miziparvovec

UX701

Gene Therapy Target: ATP7B

Wilson Disease Phase 2 intravenous NCT04884815

setrusumab

UX143

Protein Target: sclerostin

Osteogenesis Imperfecta Phase 3 intravenous Mereo NCT03118570

UX016

GNE Myopathy Preclinical subcutaneous NCT07511556

Biotech Distilled coverage (12)

2026-09-17 UPDATE: Ultragenyx (RARE) - The Last Card Flipped, and It Was Better Than the Deck Promised 🔒 Paid
2026-09-02 UPDATE: Ultragenyx (RARE) - The Crown Jewel Broke 🔒 Paid
2026-08-19 UPDATE: Ultragenyx (RARE) - Bedford Passed 🔒 Paid
2026-08-12 UPDATE: Ultragenyx (RARE) - One of My Three Sell Triggers Just Got Defused 🔒 Paid
2026-08-05 UPDATE: Ultragenyx (RARE) - The Burn Rate Just Halved, and the Angelman Clock Got a Month 🔒 Paid
2026-05-06 UPDATE: Ultragenyx (RARE) - The Angelman Safety Signal Just Got Quieter, and the Cash Got Lighter 🔒 Paid
2026-04-27 UPDATE: Ultragenyx (RARE) - Both PDUFA Dates Are Now on the Calendar 🔒 Paid
2026-03-14 UPDATE: Ultragenyx (RARE) - The Science Delivers (Again), But the Factory Remains the Boss 🔒 Paid
2026-03-01 UPDATE: Ultragenyx (RARE) - A Date With Destiny 🔒 Paid
2026-02-21 UPDATE: Ultragenyx (RARE) - 10-K Autopsy: Suing the CDMO and Mortgaging the Crown Jewels 🔒 Paid
2026-02-14 Update: Ultragenyx (RARE) - Q4 2025 Report 🔒 Paid
2026-02-09 Ultragenyx Pharmaceuticals (RARE): Scientific Deep Dive for Lead and Pipeline Products

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