FDA Approves First Treatment for Niemann-Pick Disease Type C
Zevra Therapeutics said it expects the therapy to be commercially available in the United States in 8 to 12 weeks.
The FDA has approved arimoclomol (Miplyffa) for the treatment of Niemann-Pick disease type C (NPC) in adult and pediatric patients aged 2 years and older, Zevra Therapeutics announced in a release.1 The medication is indicated for use in combination with miglustat to treat neurological manifestations of the disease.
FDA Approves First Treatment for Niemann-Pick Disease Type C / Grandbrothers - stock.adobe.com
The approval of arimoclomol was based on data from a phase 2/3 trial (NCT02612129), in which the therapy met its primary outcome measure of change in 5-domain NPC Clinical Severity Scale (NPCCSS) score from baseline to 12 months. The new drug application for arimoclomol was also supported by both additional clinical and nonclinical confirmatory evidence and FDA-preferred analyses. Results from the phase 2/3 trial were published in the Journal of Inherited Metabolic Disease.2
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“NPC is a serious disease that leads to enormous adverse impacts on patients and families. Despite extensive research efforts, there have not been approved treatments to meet the significant needs of patients,” Janet Maynard, MD, MHS, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research, said in a release.3 “The first-ever approval of a safe and effective drug option for NPC will undoubtedly support the essential medical needs of those suffering.”
Arimoclomol is an orally delivered, first-in-class investigational medication that increases the activation of transcription factors EB (TFEB) and E3 (TFE3), which results in the upregulation of coordinated lysosomal expression and regulation genes. The therapy was previously granted Breakthrough Therapy, Rare Pediatric Disease, Orphan Drug, and Fast Track designations by the FDA.
The trial supporting arimoclomol’s NDA was a prospective, randomized, double-blind, placebo-controlled phase 2/3 study evaluating the efficacy and safety of arimoclomol as an add-on therapy to the participants’ currently prescribed best routine clinical care. The study cohort included 50 patients aged 2 to 19 years who randomly received 31 to 124 mg of arimoclomol depending on weight or placebo 3 times per day for 12 months.
Of 39 patients who received arimoclomol as background treatment, disease progression as measured by the NPCCSS score was slower compared to those who received placebo. Additional confirmatory evidence from a 48-month open-label extension study also showed that arimoclomol demonstrated improved outcomes when compared to a matched National Institutes of Health NPC natural history cohort.
Adverse events occurred in 88.2% of patients who received arimoclomol and 75% of patients who received placebo. However, fewer patients in the arimoclomol group had serious adverse events compared to those in the placebo group. The most common adverse event was vomiting, and upper respiratory tract infection and decreased weight occurred more frequently with arimoclomol.
In the news release, Zevra said that it plans to immediately initiate its launch activities for arimoclomol. The company expects the therapy to be commercially available in the United States in 8 to 12 weeks.
“The FDA approval of Miplyffa marks a significant moment for those living with NPC and the global NPC community,” Elizabeth Berry-Kravis, professor in the Departments of Pediatrics, Neurological Sciences, Anatomy and Cell Biology Rush University Medical Center, said in a release.1 “Effective management of NPC requires multiple treatment options due to the complexity of the disease. Until today, there were no approved therapies in the U.S. for NPC. With this labeled indication, patients will now have more access to treatments to tackle this devastating disease.”
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References
1. Zevra Therapeutics’ MIPLYFFA™ (arimoclomol) Receives U.S. FDA Approval as Treatment for Niemann-Pick Disease Type C. News Release. Zevra Therapeutics. September 20, 2024. Accessed September 20, 2024. https://investors.zevra.com/news-releases/news-release-details/zevra-therapeutics-miplyffatm-arimoclomol-receives-us-fda
2. Mengel E, Patterson MC, Da Riol RM, et al. Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment. J Inherit Metab Dis. 2021 Nov;44(6):1463-1480. doi: 10.1002/jimd.12428. Epub 2021 Sep 7. PMID: 34418116; PMCID: PMC9293014.
3. FDA Approves First Treatment for Niemann-Pick Disease, Type C. News Release. FDA. September 20, 2024. Accessed September 20, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-niemann-pick-disease-type-c
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