The US Food and Drug Administration (FDA) has approved IntraBio’s Aqneursa (levacetylleucine) to treat neurological symptoms associated with Niemann-Pick disease type C (NPC) in adults and paediatric patients weighing at least 15kg.
Affecting approximately one in every 100,000 to 120,000 births, NPC is a rare lysosomal storage disorder caused by mutations in the NPC1 or NPC2 genes and characterised by the inability to properly metabolise lipids.
Patients typically experience systemic, neurological and psychiatric symptoms that can be debilitating and significantly impact their functional abilities.
Taken orally up to three times per day, Aqneursa is now the second drug to be approved by the FDA for NPC, following its recent approval of Zevra Therapeutics’ Miplyffa (arimoclomol).
The US regulator’s latest decision was supported by data from the two-period IB1001-301study, which enrolled 60 patients aged four years and older with a confirmed diagnosis of NPC and at least mild disease-related neurological symptoms.
Results from the study showed that Aqneursa significantly improved neurological signs and symptoms, and demonstrated functional benefits important to everyday life that were evident within 12 weeks.
Mallory Factor, IntraBio’s president and chief executive officer, said: “Patients and families in the NPC community have long awaited an effective, FDA-approved treatment, and we are proud to bring hope to those affected by this devastating disease.”
Factor added that Aqneursa “may hold potential” for treating other neurodegenerative and neurodevelopmental disorders, and that the company will continue to “rapidly develop” the drug for these additional indications.
The authorisation came just days after the FDA approved Zevra’s Miplyffa in combination with the enzyme inhibitor miglustat to treat NPC in adults and children aged two years and older.
Janet Maynard, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research, said at the time of the announcement: “Despite extensive research efforts, there have not been approved treatments to meet the significant needs of [NPC] patients.
“The first-ever approval of a safe and effective drug option for NPC will undoubtedly support the essential medical needs of those suffering.”