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Ultragenyx Pharmaceutical Inc.

RARE Rare disease Lead: Approved 7 Biotech Distilled articles ↓

Cardiovascular GI/Hepatology Metabolic Neurology Gene Therapy Nucleic Acid Protein Small Molecule

Most recent annual filing: 2026-02-15

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

Dojolvi

triheptanoin

Long-Chain Fatty Acid Oxidation Disorders Approved oral

Evkeeza

evinacumab

Protein Target: ANGPTL3

Homozygous Familial Hypercholesterolemia Approved intravenous Regeneron

rebisufligene etisparvovec

UX111

Gene Therapy Target: SGSH

Sanfilippo Syndrome Type A NDA Submitted intravenous Abeona

pariglasgene brecaparvovec

DTX401

Gene Therapy Target: G6Pase-α

Glycogen Storage Disease Type Ia NDA Submitted intravenous

apazunersen

GTX-102

Nucleic Acid Target: UBE3A antisense

Angelman Syndrome Phase 3 intrathecal

avalotcagene ontaparvovec

DTX301

Gene Therapy Target: OTC gene

Ornithine Transcarbamylase Deficiency Phase 3 intravenous

rivunatpagene miziparvovec

UX701

Gene Therapy Target: ATP7B

Wilson Disease Phase 3 intravenous

setrusumab

UX143

Protein Target: sclerostin

Osteogenesis Imperfecta Phase 3 intravenous Mereo

UX016

GNE Myopathy Phase 1 subcutaneous

Biotech Distilled coverage (7)

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 deep dives are subscriber-only.