Zevra Therapeutics Submits Marketing Authorization Application to European Medicines Agency to Review Arimoclomol for the Treatment of Niemann-Pick Disease Type C
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 惭滨笔尝驰贵贵础庐 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) ha presentato una domanda di Autorizzazione al Marketing (MAA) all'Agenzia Europea per i Medicinali (EMA) per arimoclomol, destinato al trattamento della Malattia di Niemann-Pick di tipo C (NPC). Il farmaco, commercializzato negli Stati Uniti come 惭滨笔尝驰贵贵础庐, 猫 l'unico trattamento che affronta direttamente la patologia di base della NPC migliorando l'eliminazione dei lipidi tramite l'aumento dell'espressione genica.
La domanda 猫 supportata da dati clinici approfonditi raccolti da oltre 270 pazienti con NPC, inclusi studi di Fase 2/3, uno studio di estensione a etichetta aperta, programmi di accesso ampliato e uno studio pediatrico, che coprono un'esperienza clinica di 5-7 anni. Attualmente, 89 pazienti europei sono iscritti al Programma di Accesso Ampliato dell'azienda al secondo trimestre.
Zevra Therapeutics (NASDAQ:ZVRA) ha presentado una Solicitud de Autorizaci贸n de Comercializaci贸n (MAA) a la Agencia Europea de Medicamentos (EMA) para arimoclomol, dirigido al tratamiento de la Enfermedad de Niemann-Pick Tipo C (NPC). El medicamento, comercializado en EE.UU. como 惭滨笔尝驰贵贵础庐, es el 煤nico tratamiento que aborda directamente la patolog铆a subyacente de la NPC al mejorar la eliminaci贸n de l铆pidos mediante el aumento de la expresi贸n g茅nica.
La solicitud est谩 respaldada por datos cl铆nicos extensos de m谩s de 270 pacientes con NPC, que incluyen ensayos de Fase 2/3, un estudio de extensi贸n abierto, Programas de Acceso Ampliado y un subestudio pedi谩trico, representando una experiencia cl铆nica de 5 a 7 a帽os. Actualmente, 89 pacientes europeos est谩n inscritos en el Programa de Acceso Ampliado de la empresa al segundo trimestre.
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鞚� 鞁犾箔靹滊姅 2/3靸� 鞛勳儊鞁滍棙, 瓿店皽 霛茧波 鞐办灔 鞐瓣惮, 頇曤寑 鞝戧芳 頂勲攴鸽灗, 靻岇晞 頃橃渼 鞐瓣惮毳� 韽暔頃橃棳 270氇� 鞚挫儊鞚� NPC 頇橃瀽搿滊秬韯� 靾橃霅� 甏戨矓鞙勴暅 鞛勳儊 雿办澊韯半ゼ 旮半皹鞙茧 頃橂┌, 5~7雲勳棎 瓯胳箿 頇橃瀽 瓴巾棙鞚� 氚橃榿頃╇媹雼�. 順勳灛 2攵勱赴 旮办鞙茧 89氇呾潣 鞙犽熃 頇橃瀽臧� 須岇偓鞚� 頇曤寑 鞝戧芳 頂勲攴鸽灗鞐� 霌彪霅橃柎 鞛堨姷雼堧嫟.
Zevra Therapeutics (NASDAQ:ZVRA) a soumis une demande d'autorisation de mise sur le march茅 (MAA) 脿 l'Agence europ茅enne des m茅dicaments (EMA) pour l'arimoclomol, destin茅 au traitement de la maladie de Niemann-Pick de type C (NPC). Le m茅dicament, commercialis茅 aux 脡tats-Unis sous le nom de 惭滨笔尝驰贵贵础庐, est le seul traitement qui cible directement la pathologie sous-jacente de la NPC en am茅liorant l'茅limination des lipides gr芒ce 脿 une augmentation de l'expression g茅nique.
Cette soumission est appuy茅e par des donn茅es cliniques 茅tendues provenant de plus de 270 patients atteints de NPC, incluant des essais de phase 2/3, une 茅tude d'extension en ouvert, des programmes d'acc猫s 茅largi et une sous-茅tude p茅diatrique, repr茅sentant une exp茅rience patient de 5 脿 7 ans. Actuellement, 89 patients europ茅ens sont inscrits au programme d'acc猫s 茅largi de la soci茅t茅 au deuxi猫me trimestre.
Zevra Therapeutics (NASDAQ:ZVRA) hat einen Zulassungsantrag (MAA) bei der Europ盲ischen Arzneimittel-Agentur (EMA) f眉r Arimoclomol eingereicht, das zur Behandlung der Niemann-Pick-Krankheit Typ C (NPC) entwickelt wurde. Das in den USA unter dem Namen 惭滨笔尝驰贵贵础庐 vertriebene Medikament ist die einzige Behandlung, die direkt die zugrunde liegende Pathologie von NPC anspricht, indem es die Lipidbeseitigung durch gesteigerte Genexpression verbessert.
Der Antrag st眉tzt sich auf umfangreiche klinische Daten von 眉ber 270 NPC-Patienten, darunter Phase-2/3-Studien, eine Open-Label-Verl盲ngerungsstudie, Expanded Access-Programme und eine p盲diatrische Unterstudie, die eine Erfahrung von 5-7 Jahren mit Patienten repr盲sentieren. Derzeit sind 89 europ盲ische Patienten im Expanded Access-Programm des Unternehmens im zweiten Quartal eingeschrieben.
- None.
- Regulatory approval from EMA still pending and not guaranteed
Insights
Zevra's EMA submission for arimoclomol represents significant expansion potential for their rare disease treatment already approved in the US.
Zevra Therapeutics has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for arimoclomol (marketed as 惭滨笔尝驰贵贵础庐 in the US) to treat Niemann-Pick Disease Type C (NPC). This regulatory submission represents a critical milestone in the company's global commercialization strategy for a drug that received US approval last year.
The submission is backed by extensive clinical evidence spanning 5-7 years of patient experience across more than 270 NPC patients through various clinical programs. This constitutes the most comprehensive clinical development program in NPC to date, strengthening the application's chances of approval.
Importantly, Zevra already has 89 patients enrolled in Europe through their Expanded Access Program as of Q2, establishing a foundational presence in the European market ahead of potential approval. The EMA's centralized marketing authorization procedure means that approval would grant market access across all EU Member States plus Iceland, Liechtenstein, and Norway simultaneously.
NPC is an ultra-rare genetic disorder affecting lipid metabolism, causing neurological deterioration. Arimoclomol's mechanism targets the underlying disease pathology by enhancing gene expression to improve lipid clearance - a differentiated approach in this therapeutic area. The Orphan Medicinal Product designation from the EMA also provides certain benefits including market exclusivity if approved.
European approval would significantly expand Zevra's commercial footprint and revenue potential for 惭滨笔尝驰贵贵础庐, particularly important for rare disease therapeutics where maximizing global market access is essential for commercial success and broader patient benefit.
CELEBRATION, Fla., July 28, 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 diseases, announced the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the evaluation of arimoclomol for the treatment of Niemann-Pick Disease Type C (NPC). NPC is an ultra-rare, neurological disease caused by genetic mutations that result in lipid accumulation in cells, leading to visceral, neurological, and psychiatric symptoms. Arimoclomol is the only treatment shown to directly target the underlying pathology of NPC by increasing gene expression for improved lipid clearance. Arimoclomol for the treatment of NPC has been designated as an Orphan Medicinal Product by the EMA. Arimoclomol is marketed in the U.S. under the brand name 惭滨笔尝驰贵贵础庐.
鈥淭his EMA submission marks a significant milestone for the Company as we continue to expand access to 惭滨笔尝驰贵贵础庐 in NPC patients across the globe,鈥� said Neil F. McFarlane, Zevra鈥檚 President and Chief Executive Officer. 鈥淐oncurrently, we continue to advance our global Expanded Access Program, with 89 patients enrolled in Europe at the end of Q2, reinforcing MIPLYYFA鈥檚 potential to serve as a foundational treatment option across European markets. We extend our deepest gratitude to everyone involved 鈥� especially those patients and clinicians who participated in our clinical development programs and look forward to potential European approval of arimoclomol to expand on our approval in the U.S. last year.鈥�
The EMA will review the application under the centralized marketing authorization procedure. If a marketing authorization is granted by the European Commission, the authorization is valid in all EU Member States as well as in the European Economic Area (EEA) countries Iceland, Liechtenstein and Norway.
Adrian Quartel, M.D., Zevra鈥檚 Chief Medical Officer added, 鈥淭he extensive data generated for arimoclomol has shown long-term, meaningful clinical outcomes with 5 to 7 years of patient experience across more than 270 NPC patients through a Phase 2/3 clinical trial, Open-Label Extension (OLE) study, Expanded Access Programs (EAP), and a pediatric sub-study, which is the most expansive clinical development program in NPC to date. We are confident in the strength of our MAA data package and look forward to our interactions with the EMA.鈥�
Zevra currently offers an EAP for NPC patients in certain European countries, and more information can be found at: .
About Arimoclomol
Arimoclomol is Zevra鈥檚 therapy for the treatment of Niemann-Pick disease type C (NPC), which was approved by the U.S. Food and Drug Administration on Sep. 20, 2024. Arimoclomol increases the activation of the transcription factors EB (TFEB) and E3 (TFE3) resulting in the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. Arimoclomol has also been shown to reduce unesterified cholesterol in the lysosomes of human NPC fibroblasts. The clinical significance of these findings is not fully understood. In the pivotal Phase 3 trial, arimoclomol halted disease progression compared to placebo over the twelve month duration of the trial when measured by the only validated disease progression measurement tool, the NPC Clinical Severity Scale. Arimoclomol has also received Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC.
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About Niemann-Pick Disease Type C (NPC)
Niemann-Pick disease type C (NPC) is an ultra-rare, progressive, and neurodegenerative lysosomal storage disorder characterized by an inability of the body to transport cholesterol and other lipids within the cell, leading to an accumulation of these substances in various cell types, including neurons. The disease is caused by mutations in the聽NPC1听辞谤听NPC2聽genes, which are responsible for making the聽NPC1听补苍诲听狈笔颁2听lysosomal proteins. Both children and adults can be affected by NPC with varying clinical presentations. Those living with NPC can lose independence due to physical and cognitive limitations, with key neurological impairments presenting in speech, cognition, swallowing, ambulation, and fine motor skills. Disease diagnosis can often take years, with disease progression being irreversible and often leading to early mortality.
About Zevra Therapeutics, Inc.
Zevra Therapeutics, Inc. is a commercial-stage rare disease company combining science, data, and patient needs to create transformational therapies for 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.
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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 promise and potential impact of our preclinical or clinical trial data; or the potential benefits of any of our products for any specific disease. 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, 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. These and other important factors are described in detail in the "Risk Factors" section of Zevra鈥檚 Annual Report on Form 10-K for the year ended December 31, 2024, filed on March 12, 2025, and Zevra鈥檚 Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, filed on May 13, 2025, and Zevra鈥檚 other filings with the SEC. 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. 聽
Zevra Contact
Nichol Ochsner
+1 (732) 754-2545
[email protected]
FAQ
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