ZVRA

Zevra Turns Profitable In 2025 On Strong Uptake Of MIPLYFFA For Niemann-Pick Disease Type C

(RTTNews) - Zevra Therapeutics, Inc. (ZVRA) has reported its fourth quarter and full-year 2025 financial results, marking a sharp turnaround from losses in 2024 as sales of its rare disease therapy MIPLYFFA accelerated.

MIPLYFFA (Arimoclomol), approved in combination with Miglustat, treats Niemann-Pick disease type C (NPC) in adults and children two years of age and older. MIPLYFFA addresses a progressive genetic disorder that disrupts the body's ability to transport cholesterol and other lipids inside cells.

For the fourth quarter of 2025, Zevra posted net revenue of $34.1 million, nearly tripling from $12.0 million in the same period of 2024.

Net income came in at $12.2 million, or $0.20 per share, compared with a net loss of $35.7 million, or $0.67 per share, a year earlier.

Growth was fueled by $26.4 million from MIPLYFFA, up from $10.1 million in Q4 2024, with additional contributions from OLPRUVA $0.4 million, up from $0.1 million in Q4 2024, and Expanded Access Program reimbursements, and royalties under the AZSTARYS license agreement.

For the full year, Zevra's net revenue climbed to $106.5 million, a more than fourfold increase from $23.6 million in 2024.

Zevra swung to a profit of $83.2 million, or $1.40 per share, versus a net loss of $105.5 million, or $2.28 per share, in the prior year. The turnaround was driven largely by MIPLYFFA, which contributed $87.4 million in 2025 compared with $10.1 million in 2024, alongside expanded access program reimbursements and royalties from partnered products.

Beyond MIPLYFFA, Zevra's portfolio includes OLPRUVA (sodium phenylbutyrate), approved for the long-term management of certain urea cycle disorders (UCDs) in adults and children weighing at least 20 kg. OLPRUVA generated $0.8 million in net revenue in 2025, up modestly from $0.1 million in 2024, reflecting gradual commercial adoption.

The company is also advancing Celiprolol, an investigational therapy for vascular Ehlers-Danlos syndrome (VEDS). Celiprolol has received Orphan Drug and Breakthrough Therapy designations from the U.S. FDA and is being studied in the Phase 3 DiSCOVER trial under a Special Protocol Assessment. Enrolment, which has previously been paused to allow for protocol refinements and regulatory alignment, resumed in 2025, with 52 patients enrolled by year-end, and regulatory discussions are underway to explore an accelerated development pathway.

The company's cash position strengthened to $238.9 million as of December 31, 2025. CEO Neil F. McFarlane highlighted the growing impact of MIPLYFFA in treating Niemann-Pick disease type C, noting that prescription uptake and market access continue to expand. Zevra has also relocated its headquarters to Boston to tap into a deeper talent pool and is preparing for regulatory milestones in Europe, where Arimoclomol is under review by the EMA.

ZVRA has traded between $6.19 and $13.16 over the past year. The stock closed Monday's trading at $9.12, up 1.56%. During the overnight trading session, the stock rose further to $10.58, up 16%.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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