Ultragenyx Reports Strong Revenue Growth and Pipeline Progress, Guiding to $630M-$660M in 2025

Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on developing and commercializing novel therapies for serious rare and ultrarare genetic diseases, recently reported its preliminary 2023 revenue results, cash and investments at year-end 2023, and provided 2024 guidance on select key financial metrics, including net cash used in operations. The company also shared updates on its clinical milestones and pipeline progress.

In 2023, Ultragenyx reported preliminary total revenue of $430-435 million, with Crysvita® revenue of $325-330 million and Dojolvi® revenue of $70-71 million. For 2024, the company expects total revenue to be between $500 million and $530 million, with Crysvita revenue of $375 million to $400 million and Dojolvi revenue of $75 million to $80 million. This represents a year-over-year increase of approximately 20% from 2023 to 2024.



The projected increase in Crysvita and Dojolvi revenues in 2024 can be attributed to several factors, including the expansion of geographic access to the company's medicines, increased demand outside the United States, successful commercialization, positive clinical data and regulatory milestones, and revenue recognition from new regions. Ultragenyx's strategic focus on rare and ultrarare genetic diseases allows it to operate within a niche market, potentially reducing competition and increasing the likelihood of reimbursement at premium prices due to the high unmet need and lack of alternative treatments.



For 2025, Ultragenyx expects total revenue to reach $630 million to $660 million, driven by revenue growth from its commercial portfolio and the potential approval and launch of new therapies. The company expects to submit up to three near-term BLAs (Biologics License Applications) and approvals in 2024, which could contribute to the projected revenue growth in 2025. These potential new therapies include setrusumab for osteogenesis imperfecta, DTX401 for glycogen storage disease type Ia, and other gene therapy programs.

Ultragenyx's financial guidance for 2025 reflects the company's strategic priorities and growth prospects by focusing on revenue growth from its commercial portfolio and the potential approval and launch of new therapies. The company expects to maintain a disciplined approach to research and development investments, managing costs while continuing to invest in its Phase 3 programs and commercial product launches.

In conclusion, Ultragenyx's strong revenue growth trajectory, pipeline progress, and strategic focus on rare and ultrarare genetic diseases position the company for continued success in the biopharmaceutical industry. The company's financial guidance for 2025 demonstrates its commitment to driving revenue growth and maintaining a disciplined approach to research and development investments, ultimately benefiting shareholders and patients alike.