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 (ZVRA) is a biopharmaceutical innovator focused on rare diseases, combining advanced data analytics with targeted clinical development. This page provides investors and healthcare stakeholders with essential updates on Zevra's therapeutic advancements, regulatory milestones, and strategic collaborations.
Access timely press releases and curated news covering clinical trial progress, FDA communications, and partnership announcements. Our repository ensures efficient tracking of Zevra's patient-centric developments in complex therapeutic areas, including late-stage trial outcomes and commercialization strategies.
Key updates on: clinical program advancements, regulatory submissions, research partnerships, and therapy commercialization efforts. Bookmark this page for streamlined access to Zevra's evolving role in addressing unmet rare disease needs through science-driven solutions.
Zevra Therapeutics (Nasdaq: ZVRA) announced on October 6, 2025 that it granted an aggregate of 38,000 stock options as inducement awards to two new employees under its 2023 Employment Inducement Award Plan.
Each award vests over four years with 25% vesting on the first anniversary of employment and the remainder vesting in three equal annual installments, subject to continued employment. The awards were approved by the Compensation Committee and granted as inducements in accordance with Nasdaq Rule 5635(c)(4). The Inducement Award Plan is reserved for individuals not previously employed by Zevra or returning after a bona fide non‑employment period.
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.
FAQ
What is the current stock price of Zevra Therapeutics (ZVRA)?
What is the market cap of Zevra Therapeutics (ZVRA)?
