Zevra Therapeutics Announces Open Label Extension Data Showing Sustained Long-Term Efficacy of MIPLYFFA for the Treatment of NPC Published in the Journal of Molecular Genetics and Metabolism
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 (NASDAQ:ZVRA) ha annunciato la pubblicazione dei dati sull'efficacia a lungo termine di MIPLYFFA® (arimoclomol) nel trattamento della malattia di Niemann-Pick di tipo C (NPC) sulla rivista Molecular Genetics and Metabolism.
Lo studio di estensione in aperto ha dimostrato che MIPLYFFA mantiene la sua capacità di ridurre la progressione della malattia per almeno 5 anni nei pazienti con NPC. I dati includono risultati di sicurezza ed efficacia provenienti da oltre 270 pazienti in tutto il mondo, con alcuni pazienti trattati fino a sette anni. I risultati confermano quelli del precedente trial di Fase 2/3, che aveva evidenziato l'efficacia di MIPLYFFA nel bloccare la progressione della malattia rispetto al placebo.
Lo studio ha utilizzato la scala di gravità clinica NPC a 5 domini e la versione ricalibrata a 4 domini, l'unica misura validata per la progressione della NPC, confermando benefici duraturi senza nuove preoccupazioni di sicurezza.
Zevra Therapeutics (NASDAQ:ZVRA) anunció la publicación de datos de eficacia a largo plazo de MIPLYFFA® (arimoclomol) en el tratamiento de la enfermedad de Niemann-Pick tipo C (NPC) en la revista Molecular Genetics and Metabolism.
El estudio de extensión abierto demostró que MIPLYFFA mantiene su capacidad para reducir la progresión de la enfermedad durante al menos 5 años en pacientes con NPC. Los datos incluyen resultados de seguridad y eficacia de más de 270 pacientes en todo el mundo, algunos de los cuales recibieron tratamiento hasta por siete años. Los resultados coinciden con el ensayo previo de Fase 2/3 que mostró la efectividad de MIPLYFFA para detener la progresión de la enfermedad comparado con placebo.
El estudio utilizó la escala clínica de severidad NPC de 5 dominios y la versión reajustada de 4 dominios, la única medida validada para la progresión de NPC, confirmando beneficios sostenidos sin nuevas preocupaciones de seguridad.
Zevra Therapeutics (NASDAQ:ZVRA)는 Molecular Genetics and Metabolism 저널에 MIPLYFFA® (arimoclomol)의 니만픽병 C형(NPC) 치료에 대한 장기 효능 데이터를 발표했다고 밝혔습니다.
오픈라벨 연장 연구에서 MIPLYFFA는 NPC 환자에서 최소 5년간 질병 진행을 감소시키는 능력을 유지하는 것으로 나타났습니다. 이 데이터는 전 세계 270명 이상의 환자에 대한 안전성과 효능 결과를 포함하며, 일부 환자는 최대 7년간 치료를 받았습니다. 결과는 위약 대비 질병 진행을 멈추는 MIPLYFFA의 효과를 보여준 이전 2/3상 시험과 일치합니다.
연구는 NPC 진행을 평가하는 유일한 검증된 척도인 5도메인 및 재점수화된 4도메인 NPC 임상 중증도 척도를 사용하여 지속적인 이점을 확인했으며, 새로운 안전성 문제는 발견되지 않았습니다.
Zevra Therapeutics (NASDAQ:ZVRA) a annoncé la publication de données d'efficacité à long terme concernant MIPLYFFA® (arimoclomol) dans le traitement de la maladie de Niemann-Pick de type C (NPC) dans la revue Molecular Genetics and Metabolism.
L'étude en extension ouverte a démontré que MIPLYFFA conserve sa capacité à réduire la progression de la maladie pendant au moins 5 ans chez les patients atteints de NPC. Les données comprennent les résultats de sécurité et d'efficacité de plus de 270 patients dans le monde, certains ayant été traités pendant jusqu'à sept ans. Les résultats sont cohérents avec l'essai de phase 2/3 précédent montrant l'efficacité de MIPLYFFA pour arrêter la progression de la maladie par rapport au placebo.
L'étude a utilisé l'échelle de gravité clinique NPC à 5 domaines et la version recalculée à 4 domaines, la seule mesure validée de la progression de la NPC, confirmant des bénéfices durables sans nouvelles préoccupations de sécurité.
Zevra Therapeutics (NASDAQ:ZVRA) gab die Veröffentlichung von Langzeitwirksamkeitsdaten für MIPLYFFA® (Arimoclomol) bei der Behandlung der Niemann-Pick-Krankheit Typ C (NPC) in der Fachzeitschrift Molecular Genetics and Metabolism bekannt.
Die offene Verlängerungsstudie zeigte, dass MIPLYFFA seine Fähigkeit beibehält, die Krankheitsprogression bei NPC-Patienten für mindestens 5 Jahre zu verlangsamen. Die Daten umfassen Sicherheits- und Wirksamkeitsergebnisse von über 270 Patienten weltweit, wobei einige Patienten bis zu sieben Jahre behandelt wurden. Die Ergebnisse stimmen mit der vorherigen Phase-2/3-Studie überein, die die Wirksamkeit von MIPLYFFA bei der Hemmung des Krankheitsverlaufs im Vergleich zu Placebo zeigte.
Die Studie verwendete die 5- und neu bewertete 4-Domänen-NPC-Clinical-Severity-Scale, das einzige validierte Messinstrument für den NPC-Verlauf, und bestätigte anhaltende Vorteile ohne neue Sicherheitsbedenken.
- Demonstrated sustained efficacy in reducing NPC disease progression for at least 5 years
- Safety and efficacy data from over 270 patients worldwide
- Some patients successfully treated for up to 7 years
- No new safety concerns identified in long-term treatment
- None.
Insights
MIPLYFFA shows sustained efficacy over 5 years for treating NPC, strengthening Zevra's position in the rare disease market.
Zevra Therapeutics has published compelling long-term data for MIPLYFFA (arimoclomol) in treating Niemann-Pick disease type C (NPC), a rare and fatal neurodegenerative disorder. The 48-month open-label extension (OLE) study results, published in Molecular Genetics and Metabolism, demonstrate sustained efficacy for at least 5 years, with some patients showing benefits for up to 7 years.
The data validates MIPLYFFA's ability to halt disease progression over extended periods, building upon the positive results from their previous 12-month pivotal trial. Critically, the efficacy measurements used the 5- and rescored 4-domain NPC Clinical Severity Scale, which is the only validated measurement tool for NPC progression.
For context, NPC is characterized by lysosomal dysfunction that leads to progressive neurodegeneration and is ultimately fatal. Before MIPLYFFA, treatment options were extremely limited. The drug's mechanism appears to address the underlying lysosomal dysfunction rather than just managing symptoms.
The safety profile remains favorable with no new safety concerns identified despite the extended treatment duration. The comprehensive dataset now includes over 270 patients worldwide, representing a substantial evidence base for a rare disease treatment where large clinical trials are often challenging to conduct.
This publication strengthens MIPLYFFA's clinical profile and could potentially expand its usage in the NPC patient community, representing a significant advancement in the treatment landscape for this devastating condition.
CELEBRATION, Fla., July 16, 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, announced the publication of “Long-term Efficacy and Safety of Arimoclomol in Niemann-Pick Disease Type C: Final Results of the Phase 2/3 NPC-002 48-month Open-label Extension Trial” in the peer-reviewed journal, Molecular Genetics and Metabolism (https://doi.org/10.1016/j.ymgme.2025.109189). MIPLYFFA® (arimoclomol) is an approved treatment for Niemann-Pick disease type C (NPC), a neurodegenerative disease caused by lysosomal dysfunction.
“NPC is a debilitating and ultimately fatal disease,” said Adrian Quartel, M.D., FFPM, Zevra’s Chief Medical Officer. “With MIPLYFFA, we have demonstrated its ability to halt disease progression through twelve months in our pivotal, double-blind, randomized, placebo-controlled study. For those patients who are continuing to receive treatment through the open-label extension study, we have also shown that MIPLYFFA’s clinically-meaningful impact on disease progression is sustained over multiple years, with safety and efficacy data extending through five years in more than 270 patients worldwide, and in some patients as long as seven years on treatment. This impact on disease progression represents a critical advancement in care for people living with NPC, and we are humbled by the opportunity to support this community.”
This paper presents long-term efficacy and safety outcomes for NPC patients treated with MIPLYFFA in the 48-month open-label extension (OLE) phase following the end of the 12-month, pivotal, placebo-controlled, double-blind, Phase 2/3 trial. Efficacy was calculated using the 5- and rescored 4-domain NPC Clinical Severity Scale, the only validated measurement of NPC progression. The data from the OLE phase showed a sustained reduction in disease progression for at least 5 years in a heterogeneous population of NPC patients receiving MIPLYFFA in addition to routine clinical care, with no new safety concerns. These results align with the pivotal Phase 2/3 trial, which showed that MIPLYFFA halted disease progression compared to placebo over the one-year duration.
About MIPLYFFA® (arimoclomol)
MIPLYFFA (arimoclomol) is Zevra’s approved therapy for use in combination with miglustat for the treatment of Niemann-Pick disease type C (NPC). Approved by the U.S. Food and Drug Administration on Sep. 20, 2024, MIPLYFFA (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. MIPLYFFA 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, MIPLYFFA halted disease progression compared to placebo over the one-year duration of the trial when measured by the only validated disease progression measurement tool, the NPC Clinical Severity Scale. MIPLYFFA has also received Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC.
INDICATIONS AND USAGE
MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older.
IMPORTANT SAFETY INFORMATION
Hypersensitivity Reactions:
Hypersensitivity reactions such as urticaria and angioedema have been reported in patients treated with MIPLYFFA during Trial 1: two patients reported both urticaria and angioedema (
Embryofetal Toxicity:
MIPLYFFA may cause embryofetal harm when administered during pregnancy based on findings from animal reproduction studies. Advise pregnant females of the potential risk to the fetus and consider pregnancy planning and prevention for females of reproductive potential.
Increased Creatinine without Affecting Glomerular Function:
Across clinical trials of MIPLYFFA, mean increases in serum creatinine of
During MIPLYFFA treatment, use alternative measures that are not based on creatinine to assess renal function. Increases in creatinine reversed upon MIPLYFFA discontinuation.
The most common adverse reactions in Trial 1 (≥
Three (
To report SUSPECTED ADVERSE REACTIONS, contact Zevra Therapeutics, Inc. at toll-free phone 1-844-600-2237 or FDA at 1 800-FDA-1088 or www.fda.gov/medwatch.
Drug Interaction(s):
Arimoclomol is an inhibitor of the organic cationic transporter 2 (OCT2) transporter and may increase the exposure of drugs that are OCT2 substrates. When MIPLYFFA is used concomitantly with OCT2 substrates, monitor for adverse reactions and reduce the dosage of the OCT2 substrate.
Use in Females and Males of Reproductive Potential:
Based on animal findings, MIPLYFFA may impair fertility and may increase post-implantation loss and reduce maternal, placental, and fetal weights.
Renal Impairment:
The recommended dosage of MIPLYFFA, in combination with miglustat, in patients with an eGFR ≥15 mL/minute to <50 mL/minute is lower than the recommended dosage (less frequent dosing) in patients with normal renal function.
MIPLYFFA capsules for oral use are available in the following strengths: 47 mg, 62 mg, 93 mg, and 124 mg.
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.
For more information, please visit www.zevra.com or follow us on X and LinkedIn.
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’s Annual Report on Form 10-K for the year ended December 31, 2024, filed on March 12, 2025, and Zevra’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, filed on May 13, 2025, and Zevra’s 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.
Zevra Contact
Nichol Ochsner
+1 (732) 754-2545
nochsner@zevra.com
This press release was published by a CLEAR® Verified individual.
FAQ
What are the long-term efficacy results of MIPLYFFA (ZVRA) in treating NPC?
How long was the open-label extension study for Zevra's MIPLYFFA?
What measurement tool was used to evaluate MIPLYFFA's efficacy in NPC patients?
Were there any safety concerns identified in the long-term MIPLYFFA study?
How does the open-label extension data compare to MIPLYFFA's initial trial results?
