🇺🇸 FDA
Company

Ultragenyx

Ultragenyx Pharmaceutical

RARE pharma USA

Quick answer

Ultragenyx is a pharma company (RARE) headquartered in NOVATO, CA, USA with 24 tracked pipeline programs.

Pipeline (24)

project

,4-diaminopyridine

Polyradiculoneuropathy, Chronic Inflammatory Demyelinating

Phase 3 · small_molecule · completed

1 trials project

5 mg prednisone

Granulomatosis With Polyangiitis

Phase 3 · small_molecule · active

2 trials project

Aceneuramic Acid Extended-Release Tablets

Hereditary Inclusion Body Myopathy

Phase 3 · small_molecule · terminated

1 trials project

Adjuvant Immunomodulatory (IM) Therapy

Mucopolysaccharidosis IIIA

Phase 3 · small_molecule · active

1 trials project

Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day

Sanfilippo Syndrome

Phase 3 · small_molecule · active

1 trials project

Analatro

Latrodectism

Phase 3 · small_molecule · completed

1 trials project

Anti-thymocyte globulin

Aplastic Anemia

Phase 3 · small_molecule · completed

1 trials project

Bisphosphonate

Osteogenesis Imperfecta

Phase 3 · small_molecule · active

1 trials project

Burosumab

Hypophosphatemia

Phase 3 · mab · completed

1 trials project

DTX301

OTC Deficiency

Phase 3 · gene_therapy · active

1 trials project

DTX401

Glycogen Storage Disease Type IA

Phase 3 · gene_therapy · completed

1 trials project

Evinacumab

Homozygous Familial Hypercholesterolemia

Phase 3 · small_molecule · completed

1 trials project

Fluticasone Propionate, 880 mcg twice daily (after 6FED failure)

Eosinophilic Gastrointestinal Disorders (EGIDs)

Phase 3 · small_molecule · completed

1 trials project

GTX-102

Angelman Syndrome

Phase 3 · small_molecule · active

4 trials project

Plasma exchange

Acute Disseminated Encephalomyelitis

Phase 3 · other · active

1 trials project

Rifampicin

Multiple System Atrophy

Phase 3 · small_molecule · terminated

1 trials project

Sirolimus

Lymphangioleiomyomatosis

Phase 3 · small_molecule · completed

1 trials project

Tiratricol

Monocarboxylate Transporter 8 Deficiency

Phase 3 · small_molecule · completed

1 trials project

Triheptanoin

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

Phase 3 · small_molecule · active

1 trials project

UX003

MPS 7

Phase 3 · small_molecule · completed

1 trials project

UX003

Sly Syndrome

Phase 3 · small_molecule · completed

2 trials project

UX007

Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Phase 3 · small_molecule · terminated

2 trials project

aceneuramic acid extended-release (Ace-ER)

Hereditary Inclusion Body Myopathy

Phase 3 · small_molecule · completed

1 trials project

acyclovir

Herpes Simplex

Phase 3 · small_molecule · completed

2 trials

Drugs (2)

drug

triheptanoin

DOJOLVI

 drug

vestronidase alfa-vjbk

MEPSEVII

Patents (8)

patent

US 12358976

Methods for increasing serum concentration of phosphorous and/or 1,25-hydroxy vi

Expires Mon Jul 10 2045 00:00:00 GMT+0000 (Coordinated Universal Time)

 patent

US 12338450

Gene therapy constructs for treating Wilson disease

Expires Mon Jun 19 2045 00:00:00 GMT+0000 (Coordinated Universal Time)

 patent

US 11944658

Methods of heat inactivation of adenovirus

Expires Mon Mar 28 2044 00:00:00 GMT+0000 (Coordinated Universal Time)

 patent

US 11771748

Methods for treating tumor-induced osteomalacia

Expires Mon Sep 28 2043 00:00:00 GMT+0000 (Coordinated Universal Time)

 patent

US 11753369

Creatine prodrugs, compositions and methods of use thereof

Expires Mon Sep 07 2043 00:00:00 GMT+0000 (Coordinated Universal Time)

 patent

US 11377643

Therapeutics for glycogen storage disease type III

Expires Mon Jun 30 2042 00:00:00 GMT+0000 (Coordinated Universal Time)

 patent

US 11332438

Creatine prodrugs, compositions and methods of use thereof

Expires Mon May 12 2042 00:00:00 GMT+0000 (Coordinated Universal Time)

 patent

US 11202822

Methods for treating hypophosphatemic disorders

Expires Mon Dec 16 2041 00:00:00 GMT+0000 (Coordinated Universal Time)