Zevra 1st profitable year on strong MIPLYFFA launch, partners with GeneDx on NPC genetic testing
Zevra posts first profitable year on strong MIPLYFFA launch and partners with GeneDx on NPC genetic testing
- Q4 2025 non-GAAP EPS was $0.19 (+128% YoY) and revenue $34.1M (+184% YoY), both beating estimates.
- Full-year 2025 revenue reached $106.5m for Zevra Therapeutics across its portfolio.
- Full-year 2025 net income was $83.2m versus a $105.5m loss in 2024.
- MIPLYFFA generated $87.4m in 2025 sales, including $26.4m in Q4.
- Company estimates 161 U.S. NPC patients on MIPLYFFA, about 40–50% of diagnosed.
- Zevra and GeneDx will fund and launch a U.S. sponsored exome-based genetic testing program for suspected NPC.
- Program aims to accelerate Niemann-Pick disease type C diagnosis and potential treatment identification for eligible patients.
- Global expanded access reimbursements were $13m in 2025, boosted by new ex-Europe distribution.
- European Medicines Agency review for arimoclomol in NPC is ongoing, with 120-day questions to be addressed soon.
- Phase III DISCOVER trial in vascular Ehlers-Danlos enrolled 52 of planned 150 patients.
- Management highlights strong refill rates but warns quarterly MIPLYFFA and EAP revenues will remain lumpy.
- Main concern: Sustainability and predictability of new patient starts and EAP demand amid inherently lumpy rare-disease markets.
- Cash, cash equivalents and investments totaled $238.9m versus $61.9m of debt at year-end 2025.
- Strong quarter, driven by accelerating MIPLYFFA adoption, expanding global NPC access, and tight operating discipline.
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