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 reports developments as a commercial-stage biopharmaceutical company focused on therapies for rare diseases. Recurring updates center on MIPLYFFA (arimoclomol), its U.S.-approved treatment for Niemann-Pick disease type C, including commercialization, patient access, regulatory activity and diagnostic-support initiatives tied to NPC.
Zevra news also covers financial results, pipeline programs in rare diseases such as vascular Ehlers-Danlos syndrome, and corporate actions that shape its portfolio. Recent company updates include the completed sale of the serdexmethylphenidate, or SDX, portfolio, including AZSTARYS and KP1077, as well as governance and executive-compensation announcements under Nasdaq rules.
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.Zevra Therapeutics (NasdaqGS: ZVRA) announced that its FDA-approved treatment MIPLYFFA® (arimoclomol) will be featured in three presentations at the National Niemann Pick Disease Foundation Conference in July 2025. MIPLYFFA is the first FDA-approved treatment for Niemann-Pick disease type C (NPC), indicated for use with miglustat in patients 2 years and older.
The presentations include clinical data showing that MIPLYFFA, combined with miglustat, halted disease progression at 12 months in pivotal trials. Long-term data from a 48-month open-label extension confirmed the treatment's effectiveness and safety profile. Additional research demonstrated MIPLYFFA's mechanism of action through upregulation of CLEAR genes and improvement of lysosomal function.
Zevra Therapeutics (NASDAQ: ZVRA) announced the final results of its 2025 Annual Meeting of Stockholders, where shareholders overwhelmingly re-elected Wendy L. Dixon, Ph.D. and Tamara A. Favorito as Class I Directors to serve until 2028. The incumbent directors received approximately 74% of the votes, defeating potential opposition nominees Travis C. Mickle and Arthur C. Regan, who would have received only 26.45% and 23.97% respectively. The opposition nominations were ultimately disregarded due to failure to meet SEC and bylaw requirements. Stockholders also approved Ernst & Young LLP as the company's independent auditors for FY2025.
Zevra Therapeutics (ZVRA) reported strong Q1 2025 financial results with net revenue of $20.4 million, primarily driven by $17.1 million from MIPLYFFA sales. The company completed the sale of its Pediatric Rare Disease Priority Review Voucher for $150 million, significantly strengthening its balance sheet to $217 million in cash and equivalents.
Commercial progress includes 13 new MIPLYFFA prescriptions in Q1 (total 122) with 38% market access coverage, and 5 new OLPRUVA patients (total 28) with 78% market access. The company reported a reduced net loss of $3.1 million ($0.06 per share) compared to $16.6 million in Q1 2024. Operating expenses were $22.8 million, with R&D expenses decreasing by $9.0 million while SG&A increased by $9.6 million due to commercial activities.
The company remains on track for MIPLYFFA's European MAA filing in H2 2025 and has enrolled 32 patients in the Phase 3 DiSCOVER trial for celiprolol.Zevra Therapeutics (NASDAQ: ZVRA), a commercial-stage company focused on rare disease therapies, has scheduled its Q1 2025 financial results announcement for Tuesday, May 13, 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. For telephone participation, U.S. callers can dial (800) 245-3047, while international participants should use +1 (203) 518-9765, with Conference ID: ZVRAQ125. A replay will be available on the company's website for 90 days following the presentation.
Zevra Therapeutics (ZVRA), a commercial-stage rare disease therapeutics company, has announced its participation in the upcoming Citizens Life Science Conference in New York. The company's executive leadership team will engage in a fireside chat on Wednesday, May 7, 2025, at 11:30 a.m. ET. Management will also be available for one-on-one meetings with registered conference attendees. Investors can access the live webcast through the "Events & Presentations" section on Zevra's investor relations website at investors.zevra.com.