Today, FDA approved Miplyffa (arimoclomol), an oral medication for the treatment of Neimann-Pick disease, type C (NPC). Miplyffa, in combination with the enzyme inhibitor miglustat, is indicated to treat neurological symptoms associated with NPC in adults and children 2 years of age and older. Miplyffa is the first drug approved by FDA to treat NPC.  

NPC is a rare genetic disease that results in progressive neurological symptoms and organ dysfunction. It is caused by changes in either the NPC1 or NPC2 gene, affecting the necessary transport of cholesterol and other lipids within a cell. As a result, these cells do not function as they should, ultimately causing organ damage. On average individuals affected by this devastating disease only live for about 13 years. 

The most common side effects of Miplyffa include upper respiratory tract infection, diarrhea and decreased weight. See full prescribing information for additional information on the safety and effectiveness of Miplyffa.