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.
GeneDx (Nasdaq: WGS) and Zevra Therapeutics announced a Sponsored Genetic Testing Program to expand access to GeneDx’s ExomeDx test for U.S. patients with suspected Niemann-Pick disease type C (NPC). Eligible patients may receive testing at no charge, with results available in as little as three weeks.
De-identified data from the program will be added to GeneDx Infinity™, aiming to improve NPC diagnosis speed and support clinical decision-making as new therapies emerge. Zevra will provide financial support for the program.
Zevra (NASDAQ: ZVRA) reported Q4 revenue of $34.1M (Q4 growth 31% QoQ) and FY2025 net revenue of $106.5M, driven by MIPLYFFA net revenue $87.4M. FY2025 net income was $83.2M ($1.40 basic, $1.35 diluted). Cash and securities totaled $238.9M at year-end.
Regulatory progress: EMA MAA under review; U.S. EAP enrolled 113 patients; Phase 3 DiSCOVER enrollment ongoing (52 patients).
Zevra Therapeutics (Nasdaq: ZVRA) appointed Justin Renz as Chief Financial Officer, effective March 9, 2026. Renz brings more than 25 years of biopharmaceutical financial leadership, experience in capital markets and commercial launches, and holds CPA credentials and multiple advanced degrees.
This leadership change positions Zevra to strengthen its financial foundation as it advances commercialization and execution of its rare disease strategy.
Zevra Therapeutics (Nasdaq: ZVRA) will report fourth quarter and full year 2025 corporate and financial results on Monday, March 9, 2026 after market close, followed by a conference call and audio webcast at 4:30 p.m. ET. The live webcast link and replay will be available on the company’s Investor Relations Events & Presentations page.
Telephone dial-ins include a U.S. number and an international number; a replay will be available for 90 days beginning about 5:30 p.m. ET.
Zevra Therapeutics (Nasdaq: ZVRA) announced that its executive leadership will present at the Citizens Life Sciences Conference in Miami Beach on March 11, 2026 at 9:00 a.m. ET.
Management will hold one-on-one meetings with registered attendees and a live webcast will be available via the Events & Presentations page on Zevra's investor website.
Zevra Therapeutics (NasdaqGS: ZVRA) presented four posters at the 22nd Annual WORLDSymposium on MIPLYFFA (arimoclomol) for Niemann-Pick Disease Type C (NPC).
Key highlights: four-year U.S. Early Access Program (EAP) data showing tolerability and disease stabilization across pediatric and adult patients, a post hoc NPC002 analysis showing statistically significant slowing versus placebo from month 3 through 12, and the first multi-year adult NPC dataset.
Zevra Therapeutics (Nasdaq: ZVRA) will ring the Nasdaq opening bell on Monday, February 9, 2026. CEO Neil F. McFarlane will deliver opening remarks at 9:24 a.m. ET followed by the bell at 9:30 a.m. ET. The ceremony is viewable via Nasdaq's live stream and on the Nasdaq MarketSite Tower in New York.
Zevra Therapeutics (Nasdaq: ZVRA) will present four abstracts on MIPLYFFA® (arimoclomol) at the 22nd Annual WORLDSymposium™ in San Diego, Feb 2-6, 2026. Four-year real-world safety and effectiveness data in Niemann-Pick disease type C (NPC) will be presented orally by Caroline Hastings, MD on Thursday, Feb 5, 2026 at 2:30 p.m. PT.
Three poster presentations on Feb 5 at 3:30 p.m. PT cover the U.S. Early Access Program (EAP), multi-year subgroup analyses, and efficacy from a randomized, placebo-controlled trial including arimoclomol plus miglustat. Zevra will host a non-CE satellite symposium on NPC diagnostics and therapeutics on Feb 5 at 12:15 p.m. PT. E-posters available via the WORLDSymposium app beginning Feb 3, 2026 at 5:00 p.m. PT, with on-demand access Feb 11–Mar 13, 2026. Zevra staff will be at booths #213 and #507 during the meeting.
Zevra Therapeutics (NASDAQ: ZVRA) said its executive leadership will present at the J.P. Morgan 44th Annual Healthcare Conference in San Francisco on January 15, 2026 at 9:45 a.m. PT. Management will also hold one-on-one meetings with registered attendees. A live webcast will be available via the company’s Investor Relations "Events & Presentations" page at investors.zevra.com.
Zevra Therapeutics (Nasdaq: ZVRA) executed an exclusive expanded access distribution agreement with Uniphar to broaden patient access to MIPLYFFA (arimoclomol) for reimbursed named-patient supply in select territories outside Europe. MIPLYFFA is FDA-approved and commercially available in the U.S.; a Marketing Authorisation Application for NPC has been validated and is under review by the European Medicines Agency. Zevra said the deal leverages Uniphar’s global distribution capabilities while the company continues U.S. commercialization and prepares for potential EU approval.