The FDA has approved arimoclomol (Miplyffa) as the first drug treatment for Niemann-Pick disease type C, a rare genetic disorder characterized by progressive neurological symptoms and organ dysfunction. This oral medication, used in combination with the enzyme inhibitor miglustat, is indicated for treating neurological symptoms in NPC patients aged 2 years and older.
Key points:
- Miplyffa is the first FDA-approved treatment for NPC.
- It is approved for use in combination with miglustat.
- The drug addresses a significant unmet medical need in a rare disease population.
- Approval was based on a randomized, double-blind, placebo-controlled trial in patients aged 2-19 years.
- Efficacy was demonstrated using the rescored 4-domain NPC Clinical Severity Scale (R4DNPCCSS).
Niemann-Pick disease type C (NPC) is caused by mutations in either the NPC1 or NPC2 gene, affecting cellular cholesterol and lipid transport. The disease typically results in a dramatically reduced life expectancy, with patients living an average of 13 years.
The clinical trial involved 50 patients randomized 2:1 to receive weight-adjusted Miplyffa or placebo, with 78% of participants receiving miglustat as background treatment. Miplyffa demonstrated efficacy by slowing disease progression as measured by the R4DNPCCSS, which assesses ambulation, speech, swallowing, and fine motor skills.
"The first-ever approval of a safe and effective drug option for NPC will undoubtedly support the essential medical needs of those suffering," stated Janet Maynard, MD, MHS, director of the FDA's Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine.
The prescribing information includes a warning for hypersensitivity reactions, including hives and angioedema. Common side effects include upper respiratory tract infection, diarrhea, and decreased weight. Miplyffa is contraindicated in pregnant women or those planning pregnancy.
Miplyffa received priority review, orphan drug, rare pediatric disease, fast track, and breakthrough therapy designations from the FDA. The approval was granted to Zevra Therapeutics.
Citation:
U.S. Food and Drug Administration. FDA approves first treatment for Niemann-Pick disease, type C. https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-niemann-pick-disease-type-c
