Zevra Announces Closing of Sale of Rare Pediatric Disease Priority Review Voucher for $150 Million
Zevra Therapeutics (ZVRA) has completed the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million in gross proceeds. The PRV was granted by the FDA in September 2024 following the approval of MIPLYFFA™ (arimoclomol), the first U.S. approved treatment for Niemann-Pick type C, an ultra-rare neurodegenerative disease.
The company reported unaudited cash, cash equivalents and investments of $68.7 million as of March 31, 2025. After the PRV sale's net proceeds of $148.3 million, the total cash position would be $217.0 million. The non-dilutive capital will support strategic priorities including the commercial launches of MIPLYFFA and OLPRUVA®, ongoing Phase 3 trial for celiprolol, and future strategic investments.
Zevra Therapeutics (ZVRA) ha completato la vendita del suo Voucher di Revisione Prioritaria per Malattie Pediatriche Rare (PRV) per 150 milioni di dollari in proventi lordi. Il PRV è stato concesso dalla FDA nel settembre 2024 dopo l'approvazione di MIPLYFFA™ (arimoclomol), il primo trattamento approvato negli Stati Uniti per la malattia di Niemann-Pick di tipo C, una malattia neurodegenerativa ultra-rara.
L'azienda ha riportato liquidità, equivalenti di liquidità e investimenti non verificati pari a 68,7 milioni di dollari al 31 marzo 2025. Dopo i proventi netti della vendita del PRV di 148,3 milioni di dollari, la posizione totale di liquidità sarebbe di 217,0 milioni di dollari. Il capitale non diluitivo supporterà le priorità strategiche, inclusi i lanci commerciali di MIPLYFFA e OLPRUVA®, il trial di Fase 3 in corso per celiprololo e futuri investimenti strategici.
Zevra Therapeutics (ZVRA) ha completado la venta de su Boleto de Revisión Prioritaria para Enfermedades Pediátricas Raras (PRV) por 150 millones de dólares en ingresos brutos. El PRV fue otorgado por la FDA en septiembre de 2024 tras la aprobación de MIPLYFFA™ (arimoclomol), el primer tratamiento aprobado en EE. UU. para la enfermedad de Niemann-Pick tipo C, una enfermedad neurodegenerativa ultra-rara.
La compañía reportó efectivo, equivalentes de efectivo e inversiones no auditadas de 68,7 millones de dólares al 31 de marzo de 2025. Después de los ingresos netos de la venta del PRV de 148,3 millones de dólares, la posición total de efectivo sería de 217,0 millones de dólares. El capital no dilutivo apoyará las prioridades estratégicas, incluidos los lanzamientos comerciales de MIPLYFFA y OLPRUVA®, el ensayo de Fase 3 en curso para celiprolol y futuras inversiones estratégicas.
제브라 테라퓨틱스 (ZVRA)는 희귀 소아 질병 우선 심사 바우처 (PRV)를 1억 5천만 달러에 판매 완료했습니다. 이 PRV는 2024년 9월 FDA에 의해 MIPLYFFA™ (아리모클로몰)의 승인 이후에 부여되었습니다. 이는 니만-픽 C형 질병, 즉 초희귀 신경퇴행성 질병에 대한 미국 최초의 승인 치료제입니다.
회사는 2025년 3월 31일 기준으로 6,870만 달러의 현금, 현금성 자산 및 투자금을 감사받지 않은 상태로 보고했습니다. PRV 판매의 순수익 1억 4,830만 달러를 포함하면 총 현금 보유액은 2억 1,700만 달러가 됩니다. 비희석 자본은 MIPLYFFA 및 OLPRUVA®의 상업적 출시, 셀리프로롤에 대한 진행 중인 3상 시험 및 향후 전략적 투자 등을 지원하는 데 사용될 것입니다.
Zevra Therapeutics (ZVRA) a finalisé la vente de son Bon de Révision Prioritaire pour les Maladies Pédiatriques Rares (PRV) pour 150 millions de dollars de recettes brutes. Le PRV a été accordé par la FDA en septembre 2024 suite à l'approbation de MIPLYFFA™ (arimoclomol), le premier traitement approuvé aux États-Unis pour la maladie de Niemann-Pick de type C, une maladie neurodégénérative ultra-rare.
L'entreprise a déclaré des liquidités, équivalents de liquidités et investissements non audités de 68,7 millions de dollars au 31 mars 2025. Après les produits nets de la vente du PRV de 148,3 millions de dollars, la position totale de liquidités serait de 217,0 millions de dollars. Le capital non dilutif soutiendra les priorités stratégiques, y compris les lancements commerciaux de MIPLYFFA et OLPRUVA®, l'essai de phase 3 en cours pour le célyprolol et les futurs investissements stratégiques.
Zevra Therapeutics (ZVRA) hat den Verkauf seines Priority Review Voucher (PRV) für seltene pädiatrische Krankheiten für 150 Millionen Dollar an Bruttoeinnahmen abgeschlossen. Der PRV wurde der FDA im September 2024 nach der Genehmigung von MIPLYFFA™ (Arimoclomol), der ersten in den USA zugelassenen Behandlung für Niemann-Pick Typ C, einer ultra-raren neurodegenerativen Erkrankung, gewährt.
Das Unternehmen berichtete zum 31. März 2025 über nicht geprüfte Zahlungsmittel, Zahlungsmitteläquivalente und Investitionen in Höhe von 68,7 Millionen Dollar. Nach den Nettoerlösen aus dem Verkauf des PRV von 148,3 Millionen Dollar würde die gesamte Liquiditätsposition 217,0 Millionen Dollar betragen. Das nicht verwässernde Kapital wird strategische Prioritäten unterstützen, einschließlich der kommerziellen Einführung von MIPLYFFA und OLPRUVA®, der laufenden Phase-3-Studie für Celiprolol und zukünftigen strategischen Investitionen.
- Secured $150 million in non-dilutive funding from PRV sale
- Strong cash position of $217 million after PRV sale proceeds
- First FDA-approved treatment for Niemann-Pick type C with MIPLYFFA
- Two commercial products in portfolio (MIPLYFFA and OLPRUVA)
- None.
CELEBRATION, Fla., April 07, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced the closing of the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of
“We have reached another key milestone with the closing on the sale of our PRV, which we received following the successful approval of MIPLYFFATM (arimoclomol) from the U.S. Food and Drug Administration (FDA),” said LaDuane Clifton, Zevra’s Chief Financial Officer. “The
Zevra was granted the PRV in September 2024 by the FDA, in connection with the FDA’s approval of MIPLYFFA, the first U.S. approved treatment for the ultra-rare neurodegenerative disease, Niemann-Pick type C.
In addition, the Company reported that available unaudited cash, cash equivalents and investments as of Mar. 31, 2025, were
About Zevra Therapeutics, Inc.
Zevra Therapeutics, Inc. is a commercial-stage company combining science, data, and patient need to create transformational therapies for rare diseases with limited or no treatment options. Our mission is to bring life-changing therapeutics to people living with rare diseases. With unique, data-driven development and commercialization strategies, the Company is overcoming complex drug development challenges to make new therapies available to the rare disease community.
For more information, please visit www.zevra.com or follow us on X and LinkedIn.
Financial Disclosure Advisory
The cash, cash equivalents and investments information provided in this press release is based on preliminary unaudited information and management estimates for the quarter ended March 31, 2025, is not a comprehensive statement of the Company’s financial results as of and for the fiscal quarter ended March 31, 2025 or any other period, and is subject to completion of the Company’s financial closing procedures. The Company’s independent registered public accounting firm has not conducted a review of and does not express an opinion or any other form of assurance with respect to this preliminary estimate.
Cautionary Note Concerning Forward-Looking Statements
This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, including without limitation statements regarding the potential benefits of any of our products or product candidates for any specific disease or at any dosage; the progress of our clinical trials, our strategic and product development objectives; the benefits of the transaction and its impact on the Company’s balance sheet or strategic goals; our financial position, including our cash, cash equivalents and investments and available resources; and the timing of any of the foregoing. Forward-looking statements are based on information currently available to Zevra and its current plans or expectations. They are subject to several known and unknown uncertainties, risks, assumptions, and other important factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. Given these risks and uncertainties, you should not place undue reliance on these forward-looking statements. These and other important factors are described in detail in the "Risk Factors" section of Zevra’s Annual Report on Form 10-K for the year ended December 31, 2024, and Zevra’s other filings with the Securities and Exchange Commission. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we cannot assure that such expectations will prove correct. These forward-looking statements should not be relied upon as representing our views as of any date after the date of this press release.
Zevra Contact
Nichol Ochsner
+1 (732) 754-2545
nochsner@zevra.com
Media Contact
Russo Partners
David Schull
+1 (858) 717-2310
david.schull@russopartnersllc.com
FAQ
How much did Zevra (ZVRA) sell its Priority Review Voucher for?
What is Zevra's (ZVRA) current cash position after the PRV sale?
What will Zevra (ZVRA) use the PRV sale proceeds for?
When did Zevra (ZVRA) receive FDA approval for MIPLYFFA?
What condition does Zevra's MIPLYFFA treat?
