FDA approves Miplyffa for Niemann-Pick disease type C, a rare neurological disorder.
The FDA has approved Miplyffa (arimoclomol) as the first treatment for Niemann-Pick disease, type C (NPC), a rare genetic disorder causing neurological symptoms and organ dysfunction. Evaluated in a 12-month trial with children and young adults aged 2 to 19, Miplyffa demonstrated efficacy by slowing disease progression. It should be used alongside miglustat. Common side effects include upper respiratory infections, diarrhea, and weight loss.
September 20, 2024
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