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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) 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.
Zevra Therapeutics (Nasdaq: ZVRA) announced on Dec 12, 2025 that it granted options to purchase an aggregate of 53,000 shares of common stock as inducement awards to three 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 in three equal annual installments, subject to continued employment.
Each award was granted as an inducement material to the individual entering employment in accordance with Nasdaq Rule 5635(c)(4). The Inducement Award Plan is reserved for individuals not previously employed or returning after a bona fide non-employment period.
Zevra Therapeutics (NasdaqGS: ZVRA) announced on December 2, 2025 the appointment of Alicia Secor to its Board of Directors and the retirement of director Wendy Dixon, PhD, effective the same date. The company said Secor brings more than 30 years of life sciences experience, including recent roles as President and CEO of Atalanta Therapeutics and prior CEO of Juniper Pharmaceuticals (acquired by Catalent in 2018), plus senior leadership at Genzyme and board roles at multiple biotech firms. Zevra’s chair and CEO each commented that Secor’s rare‑disease expertise will support strategy execution and thanked Dixon for her service.
Zevra Therapeutics (NasdaqGS: ZVRA) announced that Chief Financial Officer and Treasurer R. LaDuane Clifton will step down effective December 31, 2025 to pursue other professional opportunities. Clifton has served since 2015, and company leadership said he helped guide Zevra from development stage to a commercial-stage rare disease company and left a strong financial foundation.
The company has initiated a search for a new chief financial officer. No replacement has been named and no financial metrics or guidance changes were disclosed.
Zevra Therapeutics (Nasdaq: ZVRA) reported Q3 2025 results: net revenue $26.1M driven by MIPLYFFA $22.4M, compared with $3.7M in Q3 2024. Q3 net loss was $0.5M (−$0.01 per share) versus a $33.2M loss a year earlier. Cash, cash equivalents and securities were $230.4M as of September 30, 2025, supporting the company’s operating plan. Clinical and regulatory progress includes an EMA-validated MAA for arimoclomol (orphan designation) and ongoing Phase 3 DiSCOVER enrollment for celiprolol (44 patients enrolled). The company reduced OLPRUVA commercial effort while maintaining product availability.
Zevra Therapeutics (NasdaqGS: ZVRA) said members of its executive leadership will participate in a fireside discussion at Guggenheim's 2nd Annual Healthcare Innovation Conference in Boston on Wednesday, November 12, 2025 at 11:30 a.m. ET.
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) will report Q3 2025 corporate and financial results on Wednesday, November 5, 2025 after market close. The company will host a conference call and audio webcast the same day at 4:30 p.m. ET.
A webcast link and replay (available for 90 days) are accessible on the Events & Presentations page in the Investor Relations section at investors.zevra.com. Telephone dial-in: US: (800) 579-2543; International: +1 (785) 424-1789. Conference ID: ZVRAQ325. Replay begins at ~5:30 p.m. ET on November 5, 2025.
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.