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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 (ZVRA) has entered into an agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million in gross proceeds. The transaction is expected to close within 30-45 days, subject to customary conditions.
The PRV was awarded to Zevra in September 2024 following FDA approval of MIPLYFFA™ (arimoclomol), a treatment for neurological manifestations of Niemann-Pick disease type C in patients 2 years and older. The non-dilutive capital will support the commercial launches of MIPLYFFA™ and OLPRUVA®, along with advancing the company's rare disease pipeline.
Zevra Therapeutics (ZVRA), a commercial-stage company specializing in rare disease therapies, has scheduled its fourth quarter and full year 2024 financial results announcement for Tuesday, March 11, 2025. The company will release its 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 investor relations website, with dial-in options available for both U.S. (800-245-3047) and international (+1-203-518-9765) participants using Conference ID: ZVRAQ424. A replay of the webcast will be available for 90 days following the presentation.
Zevra Therapeutics (NASDAQ: ZVRA), a commercial-stage rare disease therapy company, has announced its participation in two major investor conferences in March 2025. The company's executive leadership team will present at the TD Cowen 45th Annual Health Care Conference on March 5, 2025, at 10:30 a.m. ET, and the 37th Annual ROTH Conference on March 17, 2025, at 1:00 p.m. PT.
Management will be available for one-on-one meetings with registered attendees at both conferences. Interested parties can access live webcasts of the presentations through the 'Events & Presentations' section on Zevra's Investor Relations website at investors.zevra.com.
Zevra Therapeutics (ZVRA) announced its participation in the 21st Annual WORLDSymposium™, where it will receive a 2025 New Treatment Award for MIPLYFFA™. Eight abstracts related to MIPLYFFA (arimoclomol) have been accepted for poster presentation, with one selected for oral presentation by Dr. Eugen Mengel.
MIPLYFFA is approved for treating neurological manifestations of Niemann-Pick disease type C (NPC) in patients 2 years and older, used in combination with miglustat. The featured presentation will discuss efficacy results from a 12-month double-blind randomized trial using the re-scored 4-domain NPC Clinical Severity Score.
The poster presentations will cover various aspects including gene expression, real-world treatment data, regulatory journey, safety profiles, and long-term efficacy. E-posters will be available to registered attendees from February 4, 2025, through the WORLDSymposium mobile app, with on-demand access extending to March 14, 2025. The symposium takes place February 3-7, 2025, in San Diego, CA.
Zevra Therapeutics (ZVRA), a commercial-stage rare disease therapeutics company, has announced its participation in two major investor conferences in February 2025. The company's executive leadership team will present at the Guggenheim Securities SMID Cap Biotechnology Conference on February 5 at 2:30 p.m. ET, and the Oppenheimer & Co. 35th Annual Healthcare Life Sciences Conference on February 12 at 3:20 p.m. ET.
Management will be available for one-on-one meetings with registered attendees at both conferences. Interested parties can access live webcasts of the presentations through the 'Events & Presentations' section on Zevra's Investor Relations website at investors.zevra.com.
Zevra Therapeutics (ZVRA) announced its upcoming addition to the Nasdaq Biotechnology Index (NBI) effective December 23, 2024, as part of the annual index reconstitution. The inclusion reflects the company's meeting of eligibility requirements, including minimum market capitalization, average daily trading volume, and biotechnology industry classification.
The NBI tracks the performance of biotechnology and pharmaceutical securities listed on Nasdaq under a modified capitalization-weighted methodology. CEO Neil F. McFarlane highlighted this as a transformational year for Zevra, noting that the index inclusion will raise the company's profile among biotech funds and portfolio managers.
Zevra Therapeutics (ZVRA) announced organizational changes as part of its transformation into a rare disease therapeutics company. The company is consolidating its development and scientific functions under CMO Adrian Quartel, who will oversee clinical development, quality assurance, and regulatory affairs.
Key changes include the departure of Chief Development Officer Christal Mickle and Chief Scientific Officer Sven Guenther in December 2024. The company is eliminating positions in CMC and Clinical Development, discontinuing in-house drug discovery activities, and closing laboratories in Iowa and Virginia. Future early R&D will be outsourced.
These changes align with Zevra's 2025 Strategic Plan focusing on late-stage clinical and commercial opportunities, built on four pillars: Commercial Excellence, Pipeline and Innovation, Talent and Culture, and Corporate Foundation.
Zevra Therapeutics (NASDAQ: ZVRA) has announced its participation in two major investor conferences in December 2024. President and CEO Neil F. McFarlane will present at the Piper Sandler 36th Annual Healthcare Conference on December 4 at 10:30 a.m. ET, and the Oppenheimer Movers in Rare Disease Summit on December 12 at 2:45 p.m. ET.
Management will be available for one-on-one meetings with registered attendees at both events. The Piper Sandler fireside chat will be available via webcast through the company's investor relations website.
Zevra Therapeutics (ZVRA) announces the commercial availability of MIPLYFFA™ (arimoclomol), the first FDA-approved treatment for Niemann-Pick disease type C (NPC). The medication is indicated for use with miglustat to treat neurological manifestations in patients 2 years and older. The company reports early adoption exceeding expectations, with product now available for shipment within their guided 8-12 week post-approval timeframe. AmplifyAssist™, Zevra's comprehensive patient support program, provides insurance coverage education, copay assistance, and therapy management counseling to facilitate patient access.
Zevra Therapeutics reported Q3 2024 financial results, highlighting the FDA approval of MIPLYFFA™ for Niemann-Pick disease type C treatment. The company reported revenue of $3.7 million and a net loss of ($33.2 million). Key developments include receiving a rare pediatric disease Priority Review Voucher and recording 90 prescription enrollment forms for MIPLYFFA, with 30% approved for reimbursement. Cash position stands at $95.5 million, expected to extend runway into 2027. The company completed a public offering raising $64.5 million and is implementing a strategic plan focused on commercial excellence, pipeline innovation, talent, and corporate foundation.