Fri Sep 20 14:14:35 UTC 2024: ## FDA Approves First Treatment for Fatal Genetic Disorder Neimann-Pick Disease Type C
**Washington, D.C.** – The U.S. Food and Drug Administration (FDA) has granted approval to Zevra Therapeutics’ drug, Miplyffa, for the treatment of Neimann-Pick disease type C (NPC), a rare and fatal genetic disorder affecting the nervous system and other organs. This marks the first-ever approved treatment for the condition, offering hope to patients with a life expectancy of only 13 years.
Miplyffa, an oral medication, will be used in combination with miglustat (Zavesca) to manage neurological symptoms in adults and children aged 2 and older. The FDA’s Janet Maynard highlighted the significance of this approval, stating that it “will undoubtedly support the essential medical needs of those suffering” from NPC.
Zevra Therapeutics acquired the drug from its original developer, Orphazyme, in 2022. The company anticipates making Miplyffa available in the U.S. within 8 to 12 weeks. While pricing has not yet been released, analyst Oren Livnat predicts an annual average net price of $500,000 to $600,000, with potential peak sales reaching $250 million in the U.S.
The FDA has issued a warning regarding potential hypersensitivity reactions, including hives and angioedema, for Miplyffa.
This breakthrough approval is a significant milestone in the fight against NPC, offering a new treatment option and hope for a better future for patients and their families.
First Piper 