Pipeline Report

Updated February 1st, 2026

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Showing results 4,129–4,136 of 4,419
Manufacturer ▼ Drug Name Indication Stage Status Route of Administration Drug Class
Ultragenyx Pharmaceutical Inc. DOJOLVI (triheptanoin) Glut1 DS patients with the movement disorder phenotype Phase 3 Trial Discontinued Oral Genetic Disorder
Ultragenyx Pharmaceutical Inc. Aceneuramic acid extended release (Ace-ER) GNE Myopathy Phase 3 Trial Discontinued Oral Genetic Disorder
Ultragenyx Pharmaceutical Inc. DTX301 - (Enh3ance) Ornithine Transcarbamylase (OTC) Deficiency Phase 3 Ongoing Intravenous Genetic Disorder
Ultragenyx Pharmaceutical Inc. DOJOLVI (triheptanoin) Glut1 DS patients with the movement disorder phenotype Phase 3 Trial Discontinued Oral Genetic Disorder
Ultragenyx Pharmaceutical Inc. Aceneuramic acid extended release (Ace-ER) GNE Myopathy Phase 3 Trial Discontinued Oral Genetic Disorder
Ultragenyx Pharmaceutical Inc. GTX-102 - (Aurora) Other Angelman syndrome genotypes Phase 3 Trial Planned Intrathecal Genetic Disorder
Ultragenyx Pharmaceutical Inc. GTX-102 - (Aurora) Angelman syndrome (AS) Phase 2/3 Ongoing Intrathecal Genetic Disorder
Ultragenyx Pharmaceutical Inc. Setrusumab (BPS-804) - (ORBIT) Osteogenesis Imperfecta Phase 2/3 Data Released Intravenous Orthopedic
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HCPCS Level II codes and descriptors are approved and maintained jointly by the alpha-numeric editorial panel (consisting of CMS, America's Health Insurance Plans, and Blue Cross and Blue Shield Association). CPT® codes and descriptions are copyright American Medical Association (AMA). All rights reserved.