Summary
- The FDA approved Miplyffa, an oral medication for Niemann-Pick disease, type C.
- Miplyffa, in combination with miglustat, is approved to treat neurological symptoms in adults and children 2 years and older.
- NPC is a rare genetic disease causing progressive neurological symptoms and organ dysfunction.
- Miplyffa is the first FDA-approved drug to treat NPC.
- Miplyffa was granted various designations by the FDA and should be taken orally according to recommended dose for body weight.
In a groundbreaking announcement, the U.S. Food and Drug Administration has just approved Miplyffa (arimoclomol), an oral medication designed to treat Niemann-Pick disease, type C (NPC). With the combination of the enzyme inhibitor miglustat, Miplyffa is now available to address the neurological symptoms associated with NPC in both adults and children aged 2 years and older. This marks a significant milestone as Miplyffa is the first drug to receive FDA approval for the treatment of NPC, a rare genetic disease that causes progressive neurological symptoms and organ dysfunction due to abnormalities in cholesterol and lipid transport within cells.
Following a successful twelve-month trial evaluating the drug’s safety and effectiveness, Miplyffa demonstrated a slower disease progression compared to a placebo, as measured by the rescored 4-domain NPC Clinical Severity Scale (R4DNPCCSS). While the medication comes with warnings for potential side effects such as hypersensitivity reactions and is not recommended for pregnant females, it has received various designations from the FDA to expedite its approval. Developed by Zevra Therapeutics, Miplyffa represents a significant advancement in addressing the critical medical needs of patients suffering from NPC.
Neurology, Pediatrics