Company profile

ULTRAGENYX PHARMACEUTICAL INC.

NASDAQ:RAREBiotechnology: Pharmaceutical Preparations

Drug pipeline (9)

Asset	Stage	Revenue	Trials	Status
DOJOLVI	approved	—	6	approved
GENGLYCOS	approved	—	0	—
MEPSEVII	approved	—	0	—
AVALOTCAGENE ONTAPARVOVEC	phase3	—	1	active
PARIGLASGENE BRECAPARVOVEC	phase3	—	2	no active trials

Revenue is the latest quarter the company reported for that product, from SEC filings; a fourth quarter is the year less nine months when no filing states one. Trial counts are of the product's ingredients, deduplicated by trial, so products sharing an ingredient share trials.

Clinical trials (41)

NCT ID	Phase	Status	Start	Completion	Sponsor name	Title
NCT04884815	phase1_2	active_not_recruiting	2021-09-27	2029-03-01	Ultragenyx Pharmaceutical Inc	A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
NCT01517880	phase2	completed	2012-05-01	2013-11-01	Ultragenyx Pharmaceutical Inc	A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy
NCT02230566	phase3	completed	2014-12-01	2016-05-01	Ultragenyx Pharmaceutical Inc	A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
NCT07157254	phase2	active_not_recruiting	2025-10-13	2030-01-01	Ultragenyx Pharmaceutical Inc	A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
NCT03118570	phase2	completed	2017-09-11	2019-10-01	Ultragenyx Pharmaceutical Inc	A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804

FDA applications (3)

Application

Approved

Type

Applicant

Products

BLA 125858

2026-08-19

BLA

ULTRAGENYX PHARMACEUTICAL, INC.

NDA 213687

2020-06-30

NDA

ULTRAGENYX PHARMACEUTICAL INC

BLA 761047

2017-11-15

BLA

ULTRAGENYX PHARAMCEUTICAL INC.