Ultragenyx Pharmaceutical Inc

Quick facts

Marketed products

• Mepsevii · Other

Phase 3 pipeline

• Antipyretic · Other
• GTX-102 · Neurology
  GTX-102 is an antisense oligonucleotide that reduces production of FUS protein by targeting FUS mRNA in patients with FUS-associated neurological disease.
• Placebo for oral corticosteroids
  A placebo contains no active pharmaceutical ingredient and produces no direct pharmacological effect.
• UX003 · Cardiovascular
  UX003 is a recombinant human monoclonal antibody that targets angiopoietin-like 3 (ANGPTL3) protein.

Phase 2 pipeline

• Adjuvant IM Therapy
• Optimized Prophylactic IM Therapy
• UX111

Phase 1 pipeline

• Prophylactic Corticosteroid Taper Regimen
• Reactive Corticosteroid Taper Regimen

Competitive intelligence

For the full portfolio CI brief — phase mix, recent regulatory actions, upcoming PDUFA dates, patent timeline, class-overlap with competitors:

• Ultragenyx Pharmaceutical Inc portfolio CI brief
• Ultragenyx Pharmaceutical Inc pipeline updates RSS