Welcome to our dedicated page for Zevra Therapeutics news (Ticker: ZVRA), a resource for investors and traders seeking the latest updates and insights on Zevra Therapeutics stock.
Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) is a commercial-stage rare disease company whose news flow centers on its therapies for serious, low-prevalence conditions and the corporate developments that support these programs. The company regularly issues updates describing progress with its approved products, clinical pipeline, regulatory interactions, and financial performance.
A major theme in Zevra’s news is the commercialization and clinical evidence for MIPLYFFA (arimoclomol), its approved therapy for Niemann-Pick disease type C (NPC). Press releases highlight pivotal and long-term data, presentations at scientific and patient-focused meetings, and steps to broaden access, such as a distribution agreement with Uniphar to enable reimbursed named patient supply for NPC patients in select territories outside Europe. These items provide insight into how Zevra is working to expand the reach of its NPC treatment.
News coverage also includes updates on OLPRUVA (sodium phenylbutyrate), Zevra’s approved treatment for certain urea cycle disorders, and on celiprolol, its investigational candidate for vascular Ehlers-Danlos syndrome. The company reports on clinical trial enrollment milestones, publication of new analyses, and recognition of its scientific work at conferences, giving investors and clinicians visibility into the evolution of its rare disease portfolio.
In addition, Zevra issues quarterly financial results and corporate updates, detailing revenue contributions from its commercial products, operating expenses, and capital resources. Governance and organizational news, such as board appointments, executive transitions, and equity inducement grants under its employment inducement award plan, are disclosed through press releases and Form 8-K filings. For anyone tracking ZVRA, this news stream offers a consolidated view of the company’s therapeutic progress, regulatory status, and corporate trajectory in the rare disease space.
Zevra Therapeutics (NASDAQ:ZVRA) has announced upcoming presentations of clinical data for its two key therapies at major scientific meetings. MIPLYFFA® (arimoclomol), the only treatment shown to halt Niemann-Pick disease type C progression at 12 months, will be featured at the INPDA Meeting in Argentina. The therapy has been administered to over 270 patients, representing the largest NPC clinical trial dataset.
Additionally, data on OLPRUVA® (sodium phenylbutyrate), a novel formulation for treating urea cycle disorders, will be presented. The presentations include an oral session at INPDA and multiple posters at the Child Neurology Society Annual Meeting, covering long-term efficacy, pediatric safety, and administration methods.
Zevra Therapeutics (NASDAQ:ZVRA) presented new positive data for MIPLYFFA® (arimoclomol) in treating Niemann-Pick disease type C (NPC) at the International Congress of Inborn Errors of Metabolism. The company showcased multiple posters highlighting the drug's efficacy and safety profile.
Key findings include: MIPLYFFA's ability to upregulate CLEAR network genes and amplify NPC1 protein levels, reduced disease progression in patients switching from placebo while on miglustat, demonstrated safety in children aged 6 to 24 months, and confirmed effectiveness in real-world settings. The company's poster detailing MIPLYFFA's mechanism of action received a Best Poster Award nomination from ICIEM.
Zevra Therapeutics (NasdaqGS: ZVRA) announced that four posters on MIPLYFFA® (arimoclomol) will be presented at the International Congress of Inborn Errors of Metabolism (ICIEM) in Kyoto, Japan, September 2-6, 2025. MIPLYFFA, approved in the U.S. for treating Niemann-Pick disease type C (NPC), will be featured in presentations including a Best Poster award winner highlighting its mechanism of action.
The presentations will showcase new data from a multi-center pediatric substudy in patients under two years old and a new prespecified efficacy analysis of patients on routine clinical care with miglustat who switched from placebo to MIPLYFFA. The drug has demonstrated long-term clinical outcomes across more than 270 NPC patients worldwide through various clinical trials and programs, representing the most extensive clinical development program in NPC to date.
Zevra Therapeutics (NASDAQ:ZVRA), a commercial-stage rare disease therapy company, has announced its participation in two major investor conferences in September 2025. The company will present at the Cantor Global Healthcare Conference on September 3 at 11:30 a.m. ET and the H.C. Wainwright 27th Annual Global Investment Conference on September 8 at 10:00 a.m. ET.
Management will be available for one-on-one meetings with registered attendees, and live webcasts of the presentations will be accessible through the Investor Relations section of Zevra's website.
Zevra Therapeutics (NASDAQ:ZVRA) reported strong Q2 2025 financial results with net revenue of $25.9 million, primarily driven by MIPLYFFA sales of $21.5 million. The company completed the sale of its Priority Review Voucher for $150.0 million, significantly strengthening its balance sheet.
Key highlights include 129 total MIPLYFFA prescriptions since launch with 52% market access coverage, and the submission of a Marketing Authorisation Application to the European Medicines Agency for arimoclomol in Niemann-Pick Disease Type C. The company reported net income of $74.7 million ($1.24 per basic share) and maintains a strong cash position of $217.7 million.
Zevra Therapeutics (NasdaqGS: ZVRA), a commercial-stage rare disease therapy company, announced its participation in the upcoming Canaccord Genuity 45th Annual Growth Conference. The company's executive team will engage in a fireside chat on August 13, 2025, at 1:00 p.m. ET in Boston, MA.
Management will also be available for one-on-one meetings with registered attendees. Investors can access the live webcast through the "Events & Presentations" section on Zevra's investor relations website at investors.zevra.com.
Zevra Therapeutics (NasdaqGS: ZVRA), a commercial-stage rare disease therapy company, has scheduled its second quarter 2025 financial results announcement for Tuesday, August 12, 2025. The company will release its corporate and financial results after market close, followed by a conference call and audio webcast at 4:30 p.m. ET.
Investors can access the webcast through Zevra's website investor relations section. The conference call can be joined via telephone using U.S. dial-in (800) 245-3047 or International +1 (203) 518-9765 with Conference ID: ZVRAQ225. A replay will be available on the company's website for 90 days following the presentation.
Zevra Therapeutics (NASDAQ:ZVRA) has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for arimoclomol, targeting the treatment of Niemann-Pick Disease Type C (NPC). The drug, marketed as MIPLYFFA® in the U.S., is the only treatment directly addressing NPC's underlying pathology by enhancing lipid clearance through increased gene expression.
The submission is supported by extensive clinical data from over 270 NPC patients, including Phase 2/3 trials, Open-Label Extension study, Expanded Access Programs, and a pediatric sub-study, representing 5-7 years of patient experience. Currently, 89 European patients are enrolled in the company's Expanded Access Program as of Q2.
Zevra Therapeutics (NASDAQ:ZVRA) announced the presentation of four posters at the 42nd Annual Meeting of the Southeastern Regional Genetics Group (SERGG) in Asheville, North Carolina. Three posters focus on MIPLYFFA® (arimoclomol) for Niemann-Pick disease type C (NPC) and one on OLPRUVA® (sodium phenylbutyrate) for urea cycle disorders.
The MIPLYFFA presentations highlight long-term efficacy data showing disease stabilization for up to 5 years in treated patients, real-world outcomes from the U.S. Expanded Access Program, and insights into the drug's mechanism of action. The OLPRUVA presentation demonstrates successful administration through gastrostomy tubes, expanding treatment options for UCD patients.
Zevra Therapeutics (NASDAQ:ZVRA) announced the publication of long-term efficacy data for MIPLYFFA® (arimoclomol) in treating Niemann-Pick disease type C (NPC) in the Molecular Genetics and Metabolism journal.
The open-label extension study demonstrated that MIPLYFFA maintains its ability to reduce disease progression for at least 5 years in NPC patients. The data encompasses safety and efficacy results from over 270 patients worldwide, with some patients receiving treatment for up to seven years. The results align with the previous Phase 2/3 trial showing MIPLYFFA's effectiveness in halting disease progression compared to placebo.
The study utilized the 5- and rescored 4-domain NPC Clinical Severity Scale, the only validated measurement of NPC progression, confirming sustained benefits with no new safety concerns.