Ultragenyx (RARE) Q1 2025: Latin America Drives 52% CRISVITA Growth, Unlocking Global Rare Disease Upside

May 6, 2025 | By EarningsIQ Research

Ultragenyx’s international execution powered a standout quarter, with Latin America and Turkey CRISVITA sales up 52% and pipeline milestones positioning the company for a pivotal year. Commercial momentum in key regions, late-stage pipeline progress, and regulatory clarity on UX111 underpin confidence in 2025 as a transformational inflection for the rare disease specialist. Investors now look to major data readouts and regulatory decisions as catalysts, with global expansion and operational discipline in focus.

Summary

• Latin America and Turkey Outperformance: CRISVITA sales in these regions surged, validating Ultragenyx’s international strategy.
• Pipeline Milestones Accelerate: Multiple late-stage clinical programs advanced, with regulatory and pivotal readouts on track.
• Regulatory Visibility Improves: FDA review of UX111 progressing on schedule, lowering near-term uncertainty.

Performance Analysis

Ultragenyx delivered a robust quarter, with total revenue reaching $139 million, up 28% year-over-year, fueled by broad-based commercial execution across its rare disease portfolio. CRISVITA, the company’s flagship therapy for XLH (X-linked hypophosphatemia, a rare bone disorder), contributed $103 million, with Latin America and Turkey accounting for $55 million and delivering standout 52% growth. This performance underscores the company’s ability to navigate complex reimbursement and market access environments, especially in emerging markets where word-of-mouth and physician-led demand are critical. North America and Europe also posted solid gains, though growth was more muted relative to the international surge.

Other products, including DEJOLVI, EVKEEZA, and MEPSEVI, maintained steady contributions, with DEJOLVI revenues of $17 million reflecting its established trajectory in both direct and named patient sales. The company reaffirmed its full-year revenue guidance of $640 to $670 million, driven by continued momentum in international markets, deeper penetration in both pediatric and adult segments, and new product launches. Operating expenses remained elevated at $282 million, reflecting ongoing investment in R&D and commercialization, but management signaled a path to lower cash burn in subsequent quarters as milestone payments subside and scale efficiencies emerge.

• International Expansion Drives Growth: CRISVITA’s 52% sales surge in Latin America and Turkey highlights the effectiveness of region-specific strategies and successful reimbursement negotiations.
• Pipeline Progress Bolsters Visibility: Late-stage programs, including UX143 and GTX102, advanced toward key data readouts, supporting the long-term growth narrative.
• Disciplined Capital Deployment: Cash usage, while seasonally high in Q1, is expected to moderate, with management reiterating a path to profitability by 2027.

Ultragenyx’s ability to deliver outsized growth in less penetrated markets, while advancing a diversified late-stage pipeline, positions the company at a strategic inflection ahead of major regulatory and clinical catalysts.

Executive Commentary

Strategic Positioning

1. Global Commercial Model Unlocks New Value Pools

Ultragenyx’s regionally tailored commercialization approach is yielding outsized returns, especially in Latin America and Turkey. Success here is driven by local reimbursement wins, physician advocacy, and word-of-mouth adoption, compensating for the lack of traditional diagnostic infrastructure. The company’s willingness to invest in local teams and navigate complex payer environments is now a proven differentiator, and this blueprint is being extended to new geographies and products.

2. Pipeline Execution Sets Up Multiple Catalysts

Late-stage clinical programs are advancing on schedule, with the Phase III ORBIT study for UX143 in osteogenesis imperfecta (OI) approaching a second interim analysis. Enrollment for GTX102 in Angelman syndrome is on track for full completion in 2025, while DTX301 for OTC deficiency and UX701 for Wilson disease are progressing toward pivotal milestones. Regulatory clarity on UX111 for Sanfilippo syndrome, with an FDA decision expected by August 18, adds near-term visibility and reduces binary risk.

3. Operational Discipline and Capital Allocation

Despite elevated operating expenses, management is guiding toward improved cash efficiency in the back half of the year. The company’s milestone-driven R&D investments are front-loaded, with expectations for declining cash burn as pipeline programs mature and commercial revenues scale. The stated goal remains GAAP profitability by 2027, with current cash reserves of $563 million providing runway through major clinical and regulatory catalysts.

4. Differentiation in Competitive Rare Disease Markets

Ultragenyx is increasingly competing in multi-asset rare disease markets, particularly in Angelman syndrome, where several ASO therapies are in development. The company’s strategy is to lead with potency, long-term data, and global access, leveraging its experience to build durable franchises even as the competitive landscape evolves.

Key Considerations

Ultragenyx’s Q1 showed the company’s ability to execute globally while managing a complex pipeline and regulatory agenda. Investors should weigh the following:

Key Considerations:

• International Leverage: Latin America and Turkey are now core growth engines, with local payer engagement and physician advocacy driving adoption beyond traditional markets.
• Pipeline Timing Risks: Multiple late-stage programs mean near-term data and regulatory events will drive valuation swings—timing precision and execution are critical.
• Cash Flow Inflection: Management expects cash usage to decline in coming quarters, but continued R&D investment and milestone payouts could pressure near-term margins.
• Regulatory Environment: The FDA’s evolving stance on accelerated approvals, especially in rare diseases, could impact timelines and market access for key assets.

Risks

Ultragenyx faces significant execution and regulatory risk, with binary clinical outcomes in late-stage programs and dependency on timely FDA review for UX111 and future BLAs. The competitive landscape in rare diseases is intensifying, with new entrants and alternative modalities. International expansion exposes the company to reimbursement delays, currency fluctuations, and operational complexity. Management’s confidence is clear, but investors should remain alert to timing slippage and the potential for uneven quarterly results due to order patterns and milestone-driven expenses.

Forward Outlook

For Q2 2025, Ultragenyx guided to:

• Continued revenue growth led by CRISVITA in Latin America and Turkey
• Key data readouts for UX143 and ongoing enrollment for GTX102

For full-year 2025, management reaffirmed guidance:

• Total revenue between $640 and $670 million
• CRISVITA revenue between $460 and $480 million
• DEJOLVI revenue between $90 and $100 million

Management highlighted that operating cash burn will decline in subsequent quarters, and that regulatory milestones for UX111 and BLA submissions for GTX401 remain on track. Major catalysts include interim and final data from pivotal studies and the FDA’s UX111 decision by August 18.

• CRISVITA international growth remains a key driver
• Late-stage pipeline data and regulatory decisions will shape the year’s trajectory

Takeaways

Ultragenyx’s global execution and pipeline progress set the stage for a pivotal 2025, but investors must monitor operational discipline and regulatory clarity as key risk factors.

• International Growth as a Value Lever: The company’s success in Latin America and Turkey demonstrates that rare disease franchises can scale beyond developed markets when local engagement and reimbursement are prioritized.
• Pipeline-Driven Catalysts: With multiple late-stage readouts and regulatory decisions ahead, the stock’s trajectory will hinge on execution and data quality across a diversified portfolio.
• Profitability Path in Focus: Management’s commitment to GAAP profitability by 2027 is credible if revenue growth persists and R&D investments moderate post-milestone periods, but cost discipline will be scrutinized.

Conclusion

Ultragenyx enters the remainder of 2025 with momentum from international commercial execution and a pipeline stacked with late-stage catalysts. The company’s ability to convert clinical and regulatory milestones into durable revenue growth, while managing operational complexity, will define its value creation narrative as the rare disease landscape evolves.

Industry Read-Through

Ultragenyx’s performance signals that rare disease therapies can achieve significant growth outside traditional U.S. and European markets, provided companies invest in local market access and physician networks. The successful navigation of reimbursement in emerging markets like Brazil and Mexico is a blueprint for peers targeting global expansion. The regulatory clarity and capital discipline demonstrated this quarter will resonate across the biotech sector, especially as late-stage gene therapy programs approach pivotal data and approval milestones. Competitors in rare diseases should note the rising bar for commercial execution and the importance of operational agility in managing complex, multi-asset pipelines.

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