The U.S. FDA has approved IntraBio's new oral drug, branded Aqneursa, for the treatment of neurological symptoms associated with Niemann-Pick disease type C (NPC), marking the second approval for the rare disease in the past week.
Aqneursa, which consists of flavored granules that are mixed with water or juice for administration, is the only FDA-approved stand-alone therapy indicated for the treatment of NPC, a rare inherited lysosomal disease that causes systemic, neurological and psychiatric symptoms that can be debilitating and significantly impact functional abilities. Just last week, Zevra Therapeutic's rival treatment, Miplyffa, was the first to get the FDA nod for NPC — however Miplyffa is designed to be taken along with the enzyme inhibitor, miglustat.
Aqneursa was approved following positive phase 3 data from a 60-person trial demonstrating significant improvements in neurological symptoms and functional benefits within 12 weeks in adult and pediatric NPC patients. Given the severity of the disease — individuals affected by NPC only live for about 13 years — the FDA granted Aqneursa Priority Review, Fast Track, Orphan Drug and Rare Pediatric Disease designations for the NPC application.