Zevra Therapeutics Reports Strong Q4 Results and 2025 Growth

Zevra Therapeutics (NASDAQ: ZVRA) announced its fourth quarter and full-year 2025 financial results, showcasing significant commercial growth for its Niemann-Pick disease type C (NPC) therapy, MIPLYFFA, and marking a transition to profitability. The company reported a net revenue of $106.5 million for the year, with MIPLYFFA contributing $87.4 million in its inaugural year of commercialization. In the fourth quarter alone, Zevra generated total net revenue of $34.1 million, which included $26.4 million from MIPLYFFA sales.

During the earnings call, President and CEO Neil McFarlane described 2025 as “a banner year” for the company, highlighting the strong commercial momentum behind MIPLYFFA. Chief Commercial Officer Josh Shafer noted that adherence rates among patients who begin therapy are robust, although he added it is still early to provide comprehensive data on long-term treatment continuity. Notably, approximately half of the patients receiving MIPLYFFA are adults, a trend consistent with findings from the company’s previous U.S. expanded access program.

Zevra’s strategy to improve diagnosis and access has contributed to its commercial success. Shafer attributed the increase in newly diagnosed patients to a “three-pronged approach,” which includes the disease awareness campaign “Learn NPC: Read between the signs,” an AI-driven targeting model, and partnerships with genetic testing providers. As of now, coverage for MIPLYFFA has reached 68% of total covered lives in the U.S., with high reimbursement rates secured for the remaining patients through dedicated market access efforts.

Zevra is also expanding access to arimoclomol (MIPLYFFA) beyond the U.S. through its global expanded access program (EAP), which enrolled 113 patients by the end of 2025. McFarlane stated that the company has built a strong reputation across Europe via this EAP and has established new distribution agreements to extend its reach beyond Europe, initiating named patient supply shipments before year-end.

In terms of regulatory developments, Zevra submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) in late July 2025. This application was supported by data from a pivotal Phase 2/3 trial, open-label extension results, and a pediatric sub-study. At the end of 2025, the company received a “120-day list of questions” from EU regulators and is preparing to respond during the allotted 90-day review period. McFarlane noted that diagnosis rates for NPC in Europe are higher than in the U.S., partly due to the approval of miglustat for NPC, which has increased physician awareness.

Beyond NPC, Zevra is developing celiprolol for vascular Ehlers-Danlos syndrome (vEDS), a rare inherited connective tissue disorder. McFarlane explained that approximately 7,500 individuals have been diagnosed with vEDS in the U.S. The ongoing Phase 3 DiSCOVER study aims to confirm clinical benefits previously observed in studies conducted outside the U.S. As of the end of 2025, Zevra has enrolled 52 patients out of a target of 150, with one confirmed event reported out of the 28 required for an interim analysis.

To enhance patient enrollment, Zevra is establishing a network of genetic testing centers and engaging with specialists such as vascular surgeons. The company has also held discussions with the FDA regarding regulatory pathways to accelerate the celiprolol program, with further engagement anticipated in the latter half of the year.

In terms of financials, Zevra reported net income of $83.2 million, or $1.40 per basic share and $1.35 per diluted share, for the full year 2025, a significant turnaround from a net loss of $105.5 million in 2024. Operating expenses for the year totaled $90.4 million, which included $12.7 million in research and development and $77.6 million in selling, general, and administrative expenses. As of December 31, 2025, the company reported $238.9 million in cash and cash equivalents, alongside approximately $61.9 million in total debt.

During the Q&A session, management emphasized that they evaluate capital allocation quarterly, focusing on U.S. commercial execution, geographic expansion, and progressing the celiprolol program. Additionally, Zevra announced leadership changes, with Justin Renz appointed as Chief Financial Officer, effective the day of the call. Renz expressed his commitment to listening, learning, and engaging with stakeholders to support the company’s execution and long-term value creation.

Zevra Therapeutics is dedicated to discovering and developing proprietary prodrugs aimed at treating serious medical conditions in the United States. The company utilizes its Ligand Activated Therapy platform, with its lead candidate, KP1077, currently undergoing clinical trials for idiopathic hypersomnia and narcolepsy, alongside the development of celiprolol for vascular Ehlers-Danlos syndrome.