Ultragenyx Announces Positive 2024 Revenue and Advances Rare Disease Pipeline
• Ultragenyx reported preliminary 2024 revenue of $555 million to $560 million, exceeding previous guidance, driven by strong sales of Crysvita and Dojolvi.
• The company anticipates total revenue between $640 million and $670 million in 2025, projecting continued growth and pipeline advancement.
• Key clinical programs are progressing, including setrusumab for osteogenesis imperfecta (Phase 3) and GTX-102 for Angelman syndrome (Phase 3).
• Ultragenyx expects a PDUFA decision for UX111 in Sanfilippo syndrome type A and plans to file a BLA for DTX401 in Glycogen Storage Disease Type Ia in 2025.
Gene Therapy Clinical Trial Pipeline Surges with Over 180 Companies Leading Innovation
• The gene therapy market is experiencing substantial growth, fueled by technological advancements, increased investments, and a deeper understanding of genetic diseases.
• Key players are actively engaging in collaborations, mergers, and acquisitions to enhance their research and development capabilities and broaden their product portfolios.
• Regulatory bodies like the FDA and EMA are expediting the approval process for gene therapies, acknowledging their potential in addressing unmet medical needs.
• Ethical considerations and public perception surrounding gene editing, particularly germline modifications, remain critical factors influencing market dynamics and regulatory policies.
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