Zevra Therapeutics (NASDAQ:ZVRA) reported second-quarter 2026 net revenue of $39.7 million, up 53% from $25.9 million a year earlier, driven primarily by U.S. sales of its Niemann-Pick disease type C treatment MIPLYFFA.
Net U.S. revenue for MIPLYFFA totaled $30.2 million during the quarter, increasing 23% from the first quarter and 40% from the prior-year period. Chief Executive Officer Neil McFarlane said the company’s commercial momentum reflects continued adoption of MIPLYFFA in combination with miglustat as a foundational treatment for NPC, a rare genetic disease.
MIPLYFFA Enrollment and Market Access
As of June 30, Zevra had received 184 prescription enrollment forms for MIPLYFFA since launch, including 14 during the second quarter and 23 during the first half of 2026. The second-quarter enrollments included patients in Puerto Rico, according to the company.
Josh Schafer, Zevra’s chief commercial officer, said enrollment forms represent prescriptions submitted to the company’s specialty pharmacy, which then begins the benefits investigation process. He said enrollments have come from both centers of excellence and community practices, and include newly diagnosed and previously diagnosed patients as well as pediatric and adult patients.
McFarlane said Zevra is seeing an approximately equal mix of adult and pediatric patients among the 184 enrollment forms. The company estimates that 900 people in the U.S. may be living with NPC, of whom 300 to 350 are diagnosed.
Schafer said Zevra’s disease-awareness efforts, genetic-testing collaborations, AI-driven physician targeting and Expert Connect program are intended to help identify patients and support diagnosis. The company’s payer coverage remained at 69% of covered lives during the quarter.
Management said refill, compliance and persistence rates have been high among patients receiving MIPLYFFA. Schafer said that, among patients who have stopped treatment, the vast majority had died as a result of the severity of the disease.
European Review of Arimoclomol
Zevra is pursuing a re-examination of its Marketing Authorization Application for arimoclomol in the European Union after the Committee for Medicinal Products for Human Use issued a negative opinion following the company’s oral explanation.
McFarlane said the re-examination will enable Zevra to address the grounds cited in the CHMP’s refusal and have its evidence package reviewed by a new rapporteur and co-rapporteur. The process typically takes 120 days from submission to a CHMP opinion, with an outcome expected in the fourth quarter.
The company has also requested a Scientific Advisory Group meeting as part of the process. McFarlane said Zevra’s confidence is based on the totality of its evidence, including its pivotal study, a four-year open-label extension study, early-access program data, a pediatric substudy and a broader safety database.
Meanwhile, Zevra will continue providing arimoclomol access for eligible patients through its global expanded-access program, which includes compassionate-use and reimbursed named-patient programs. The program had enrolled 132 patients as of June 30, including 10 added in the second quarter. McFarlane said most of those patients are in Europe.
Net reimbursements totaled $9 million in the quarter. McFarlane said the French expanded-access program has been consistent at approximately $2.5 million in net revenue per quarter, or about $10 million annually, while reimbursement and ordering patterns in other territories remain unpredictable.
Pipeline and Intellectual Property
Zevra said it secured an Orange Book listing for MIPLYFFA providing U.S. patent protection through 2041. The company is still awaiting a decision from the U.S. Patent Office on a separately requested patent-term extension. McFarlane said the longer intellectual-property protection gives Zevra a basis to consider further investments in the MIPLYFFA franchise, including potential global expansion.
The company is also advancing celiprolol in its Phase III DISCOVER trial for vascular Ehlers-Danlos syndrome. The event-driven study has enrolled 66 patients, including four during the second quarter, out of a planned 150 patients. It requires 28 events to trigger an interim analysis, and Zevra has recorded three confirmed events to date.
McFarlane said the company is working to accelerate enrollment by expanding its network of genetic-testing centers and is exploring regulatory pathways with the Food and Drug Administration. Following a Type C meeting in the first quarter, Zevra expects to reengage with the FDA during the second half of 2026.
Cash Position and Expenses
Operating expenses were $21 million in the second quarter, down $3.2 million from a year earlier. Research and development expense increased $1.1 million to $4.5 million, primarily due to ongoing arimoclomol efforts, while selling, general and administrative expense declined $4.2 million to $16.6 million, primarily because of lower professional fees and third-party spending.
Zevra ended the quarter with $260.2 million in cash equivalents and investments, up approximately $23.4 million from March 31. Chief Financial Officer Justin Renz said the company has the financial capacity to pursue MIPLYFFA growth, global opportunities, celiprolol development and selected external opportunities while maintaining disciplined capital allocation.
Renz said share repurchases are not part of Zevra’s current capital-allocation strategy, as management remains focused on growth investments and evaluating potential value-creating opportunities.
About Zevra Therapeutics (NASDAQ:ZVRA)
Zevra Therapeutics, Inc discovers and develops various proprietary prodrugs to treat serious medical conditions in the United States. The company develops its products through Ligand Activated Therapy platform. Its lead product candidate is KP1077, consisting of KP1077IH, which is under Phase 2 clinical trial for the treatment of idiopathic hypersomnia, and KP1077N, which is under Phase ½ clinical trial to treat narcolepsy. The company is also developing Celiprolol, a prodrug product candidate that is under Phase 1/2 clinical trial for the treatment of vascular Ehlers Danlos syndrome.
