FDA Approves IntraBio's Aqneursa For Niemann-Pick Disease, Type C

(RTTNews) - The U.S. Food and Drug Administration approved IntraBio Inc.'s Aqneursa (levacetylleucine) for the treatment of neurological symptoms associated with Niemann-Pick disease type C or NPC in adults and pediatric patients weighing at least 15 kilograms.

The latest decision follows the agency's last week's approval of the first treatment for NPC in adults and children 2 years of age and older, given to Zevra Therapeutics's Miplyffa (arimoclomol).

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, due to which they do not function as they should, causing organ damage. On average, individuals affected by this devastating disease only live for about 13 years.

The FDA now noted that Aqneursa's safety and efficacy for the treatment of NPC were evaluated in a randomized, double-blind, placebo-controlled, two-period, 24-week crossover study of 60 patients.

The primary efficacy outcome was a modified version of the Scale for the Assessment and Rating of Ataxia (SARA), referred to as the functional SARA (fSARA). On average, participants treated with Aqneursa for 12 weeks showed a better outcome in the fSARA score compared to when they were treated with placebo.

The use of Aqneursa may cause embryo-fetal harm if used during pregnancy. The most common side effects are abdominal pain, difficulty swallowing, upper respiratory tract infections and vomiting.

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, "This is the second treatment the FDA has approved for NPC within the span of a week. Today's action further underscores the agency's commitment to support development of new treatments for rare diseases."

The FDA granted Aqneursa Priority Review, Fast Track, Orphan Drug and Rare Pediatric Disease designations for this application.

Aqneursa is required to be taken orally up to three times per day, with or without food.

The FDA earlier noted that Miplyffa (arimoclomol), an oral medication for the treatment of NPC, was the first product application to be discussed at the inaugural meeting of the Genetic Metabolic Diseases Advisory Committee or GeMDAC in August.

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