Ultragenyx Pharmaceutical

RARE Rare disease Lead: Approved ★ Read the deep dive → 13 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

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 (13)

2026-10-08 UPDATE: Ultragenyx (RARE) - The Voucher Cashed at Double 🔒 Paid
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

Articles open on Substack. Some are subscriber-only; those marked ★ Distilled Pro are for Distilled Pro members.