Zevra Therapeutics Announces MIPLYFFA庐 (arimoclomol) Featured in Presentations at the National Niemann Pick Disease Foundation Conference
Zevra Therapeutics (NasdaqGS: ZVRA) announced that its FDA-approved treatment MIPLYFFA庐 (arimoclomol) will be featured in three presentations at the National Niemann Pick Disease Foundation Conference in July 2025. MIPLYFFA is the first FDA-approved treatment for Niemann-Pick disease type C (NPC), indicated for use with miglustat in patients 2 years and older.
The presentations include clinical data showing that MIPLYFFA, combined with miglustat, halted disease progression at 12 months in pivotal trials. Long-term data from a 48-month open-label extension confirmed the treatment's effectiveness and safety profile. Additional research demonstrated MIPLYFFA's mechanism of action through upregulation of CLEAR genes and improvement of lysosomal function.
Zevra Therapeutics (NasdaqGS: ZVRA) ha annunciato che il suo trattamento approvato dalla FDA, MIPLYFFA庐 (arimoclomol), sar脿 presentato in tre interventi alla National Niemann Pick Disease Foundation Conference nel luglio 2025. MIPLYFFA 猫 il primo trattamento approvato dalla FDA per la malattia di Niemann-Pick di tipo C (NPC), indicato per l'uso in combinazione con miglustat in pazienti di et脿 pari o superiore a 2 anni.
Le presentazioni includono dati clinici che dimostrano come MIPLYFFA, associato a miglustat, abbia interrotto la progressione della malattia a 12 mesi negli studi fondamentali. Dati a lungo termine provenienti da un'estensione in aperto di 48 mesi hanno confermato l'efficacia e il profilo di sicurezza del trattamento. Ulteriori ricerche hanno evidenziato il meccanismo d'azione di MIPLYFFA attraverso l'upregolazione dei geni CLEAR e il miglioramento della funzione lisosomiale.
Zevra Therapeutics (NasdaqGS: ZVRA) anunci贸 que su tratamiento aprobado por la FDA, MIPLYFFA庐 (arimoclomol), ser谩 presentado en tres ponencias en la Conferencia Nacional de la Fundaci贸n para la Enfermedad de Niemann-Pick en julio de 2025. MIPLYFFA es el primer tratamiento aprobado por la FDA para la enfermedad de Niemann-Pick tipo C (NPC), indicado para su uso junto con miglustat en pacientes de 2 a帽os o m谩s.
Las presentaciones incluyen datos cl铆nicos que muestran que MIPLYFFA, combinado con miglustat, detuvo la progresi贸n de la enfermedad a los 12 meses en ensayos clave. Datos a largo plazo de una extensi贸n abierta de 48 meses confirmaron la efectividad y el perfil de seguridad del tratamiento. Investigaciones adicionales demostraron el mecanismo de acci贸n de MIPLYFFA mediante la regulaci贸n al alza de los genes CLEAR y la mejora de la funci贸n lisosomal.
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氚滍憸 雮挫毄鞐愲姅 MIPLYFFA臧� 氙戈竴耄姢韮�韸胳檧 氤戩毄 鞁� 欤检殧 鞛勳儊鞁滍棙鞐愳劀 12臧滌洈 霃欖晥 歆堧硲 歆勴枆鞚� 欷戨嫧鞁滌及雼る姅 鞛勳儊 雿办澊韯�臧� 韽暔霅橃柎 鞛堨姷雼堧嫟. 48臧滌洈臧勳潣 臧滊癌順� 鞐办灔 鞐瓣惮鞚� 鞛リ赴 雿办澊韯半姅 旃橂鞚� 須臣鞕� 鞎堨爠靹� 頂勲韺岇澕鞚� 頇曥澑頄堨姷雼堧嫟. 於旉皜 鞐瓣惮鞐愳劀電� CLEAR 鞙犾爠鞛愳潣 靸來枼 臁办爤瓿� 毽唽膦� 旮半姤 臧滌劆鞚� 韱淀暅 MIPLYFFA鞚� 鞛戩毄 旮办爠霃� 鞛呾霅橃棃鞀惦媹雼�.
Zevra Therapeutics (NasdaqGS : ZVRA) a annonc茅 que son traitement approuv茅 par la FDA, MIPLYFFA庐 (arimoclomol), sera pr茅sent茅 lors de trois communications 脿 la conf茅rence de la National Niemann Pick Disease Foundation en juillet 2025. MIPLYFFA est le premier traitement approuv茅 par la FDA pour la maladie de Niemann-Pick de type C (NPC), indiqu茅 en association avec le miglustat chez les patients 芒g茅s de 2 ans et plus.
Les pr茅sentations incluront des donn茅es cliniques montrant que MIPLYFFA, associ茅 au miglustat, a arr锚t茅 la progression de la maladie 脿 12 mois lors des essais pivots. Des donn茅es 脿 long terme issues d'une extension en ouvert de 48 mois ont confirm茅 l'efficacit茅 et le profil de s茅curit茅 du traitement. Des recherches suppl茅mentaires ont d茅montr茅 le m茅canisme d'action de MIPLYFFA via la r茅gulation 脿 la hausse des g猫nes CLEAR et l'am茅lioration de la fonction lysosomale.
Zevra Therapeutics (NasdaqGS: ZVRA) gab bekannt, dass seine von der FDA zugelassene Behandlung MIPLYFFA庐 (Arimoclomol) auf drei Pr盲sentationen bei der National Niemann Pick Disease Foundation Conference im Juli 2025 vorgestellt wird. MIPLYFFA ist die erste von der FDA zugelassene Behandlung f眉r die Niemann-Pick-Krankheit Typ C (NPC) und ist f眉r die Anwendung zusammen mit Miglustat bei Patienten ab 2 Jahren zugelassen.
Die Pr盲sentationen enthalten klinische Daten, die zeigen, dass MIPLYFFA in Kombination mit Miglustat die Krankheitsprogression nach 12 Monaten gestoppt hat. Langzeitdaten aus einer 48-monatigen offenen Verl盲ngerung best盲tigten die Wirksamkeit und das Sicherheitsprofil der Behandlung. Weitere Forschungen zeigten den Wirkmechanismus von MIPLYFFA durch Hochregulierung der CLEAR-Gene und Verbesserung der lysosomalen Funktion.
- First FDA-approved treatment for Niemann-Pick disease type C
- Clinical data shows treatment halted disease progression at 12 months
- Long-term safety and effectiveness confirmed in 48-month extension study
- Treatment demonstrated clear mechanism of action through multiple pathways
- None.
Insights
Zevra's MIPLYFFA for NPC is featured at a conference, showcasing its disease-halting efficacy as the first FDA-approved treatment.
Zevra's conference presentations on MIPLYFFA represent a significant milestone in rare disease treatment. As the first FDA-approved therapy for Niemann-Pick disease type C (NPC), MIPLYFFA addresses a critical unmet need in a serious lysosomal storage disorder with limited treatment options. The pivotal study data being presented demonstrates that MIPLYFFA, when used with miglustat, halted disease progression at 12 months compared to placebo鈥攁 remarkable outcome for a progressive neurological disease.
What makes this therapy particularly noteworthy is its novel mechanism targeting the underlying disease pathophysiology. MIPLYFFA increases activation of transcription factors TFEB and TFE3, upregulating the CLEAR gene network and improving lysosomal function. The presented in vitro data confirms multiple mechanistic pathways, including increased NPC1 protein processing and enhanced cholesterol clearance from lysosomes.
The drug's 48-month open-label extension data further validates both its long-term effectiveness and safety profile, with no new safety signals observed. While the safety section notes hypersensitivity reactions in some patients and potential embryofetal toxicity, these are manageable concerns for a treatment addressing a serious progressive disease.
Zevra now has a commercial-stage product with orphan designation both in the US and Europe, creating a significant market opportunity despite the small patient population. For a rare disease therapy company, having an FDA-approved product with demonstrated efficacy transforms their commercial prospects and establishes them as a legitimate player in the orphan drug space.
CELEBRATION, Fla., July 11, 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 an oral presentation and two poster presentations on MIPLYFFA庐 (MY-PLY-FAH) (arimoclomol) are being featured at the National Niemann Pick Disease Foundation (NNPDF) Conference, taking place July 10-13, 2025, in Concord, North Carolina. The presentations review data on MIPLYFFA, the first treatment approved by the U.S. Food and Drug Administration (FDA) for the treatment of Niemann-Pick disease type C (NPC). MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older.
鈥淚 am excited to be presenting an overview of MIPLYFFA and its role in the treatment of Niemann-Pick Disease Type C,鈥� said Barbara K. Burton, M.D., Professor of Pediatrics at Northwestern University Feinberg School of Medicine and Attending Physician in the Division of Genetics, Genomics and Metabolism at the Ann & Robert H. Lurie Children's Hospital of Chicago. 鈥淣PC is a lysosomal storage disease, and MIPLYFFA鈥檚 unique mechanism improves lysosomal function, thereby addressing the underlying pathophysiology or cause of the disease. With data from a pivotal study demonstrating that MIPLYFFA, in combination with miglustat, halted disease progression at 12 months, I believe it鈥檚 one of the most important therapies available for patients with NPC.鈥�
Presentation Details
| 听 | |
| Title: | Advances in Niemann-Pick Disease Type C Treatment: The Role of Arimoclomol |
| Date/Time: | Friday, July 11, 2025; 4:00pm ET |
| Presenter: | Barbara K. Burton, M.D., Professor of Pediatrics at Northwestern University Feinberg School of Medicine and Attending Physician in the Division of Genetics, Genomics and Metabolism at the Ann & Robert H. Lurie Children's Hospital of Chicago |
Poster Details | |
| Title: | Long-Term Effectiveness and Safety Evaluation of Arimoclomol Treatment in Patients With Niemann-Pick Disease Type C 鈥� Data From the Pivotal Study and Open-Label Extension |
| Authors: | Eugen Mengel, Sven Guenther, Lauren Hitchins, Christine 铆 Dali |
| Summary: | In the pivotal trial, arimoclomol in combination with miglustat halted disease progression through 12 months compared with placebo as measured by the R4DNPCCSS. The effectiveness and safety of arimoclomol with miglustat was further confirmed in a 48-month open-label extension. Arimoclomol was generally well tolerated with no new safety signals observed during the OLE. |
| Title: | Arimoclomol Upregulates Expression of Genes Belonging to the Coordinated Lysosomal Expression and Regulation (CLEAR) Network |
| Authors: | Hadeel Shammas, Nikolaj Havns酶e Torp Petersen, Pontus Klein, Anja Koustrup, Marianne Terndrup Pedersen, Anne Sigaard Bie, Travis Mickle, Cathrine Kolster Fog, Thomas Kirkegaard Jensen, Sven Guenther |
| Summary: | The presented in vitro data provide mechanistic evidence of how arimoclomol can target NPC through multiple mechanistic pathways making it relevant in NPC. Increased translocation of the transcription factors TFE3 and TFEB from the cytosol to the nucleus is a crucial step that results in upregulation of a series of downstream processes that may improve lysosomal function and cell viability. Overall, the data support that arimoclomol does not only upregulate expression of certain CLEAR genes and specifically NPC1 at the transcriptional level, but also that this overexpression results in amplification of NPC1 protein levels and more successful NPC1 processing ultimately leading to increased cholesterol clearance from the lysosomal compartments. The effects of arimoclomol in mutant NPC cells found across the in vitro studies are consistent, and downstream effects expected to result from the activation of a specific process in one study could be confirmed in another study to provide an understanding of the mechanism of action of arimoclomol. |
About MIPLYFFA庐听(arimoclomol)
MIPLYFFA (arimoclomol) is Zevra鈥檚 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听in Trial 1 (鈮�
Three (
Call your doctor for medical advice about side effects. You may report side effects to Zevra at 1-844-600-2237, or to the FDA at 1-800-FDA-1088 or听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 鈮�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 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听NPC1听or听NPC2听genes, which are responsible for making the听NPC1听and听NPC2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 company combining science, data, and patient needs 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 or follow us on and .
Caution 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 upcoming events or Zevra鈥檚 participation at such events. 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.
Zevra Contact
Nichol Ochsner
+1 (732) 754-2545
FAQ
What is MIPLYFFA and what was it approved for by the FDA?
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