FDA approves second Niemann-Pick disease type C treatment
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The FDA has approved Aqneursa as an oral treatment for neurological symptoms associated with Niemann-Pick disease type C, a rare genetic disease, in adults and children weighing at least 15 kg, according to a press release.
Aqneursa (levacetylleucine, IntraBio Inc.) is the second treatment that the FDA has approved for Niemann-Pick disease type C (NPC) in a week. Miplyffa (arimoclomol, Zevra Therapeutics) was the first drug approved to treat the condition.
“Today’s action further underscores the agency’s commitment to support development of new treatments for rare diseases,” Janet Maynard, MD, MHS, director of the FDA’s Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, said in the release. “This approval again demonstrates the FDA’s commitment to work with the scientific community to overcome the unique challenges that may arise with rare disease drug development.”
NPC is caused by changes in NPC1 or NPC2 genes that affect the transfer of cholesterol and other lipids within cells, leading to neurological symptoms and organ dysfunction, according to the release. The average lifespan of individuals affected by NPC is about 13 years.
Levacetylleucine should be administered orally up to three times each day, according to the FDA. Its approval is based on data from a randomized, double-blind, placebo-controlled, two-period, 24-week crossover study. The study included 60 patients aged 4 years or older with NPC and at least mild neurological symptoms associated with the disease. The patients received 12 weeks of placebo and 12 weeks of levacetylleucine. They were also able to receive miglustat as background therapy.
Compared with placebo, the FDA said levacetylleucine was associated with improved function, as measured by a modified version of the Scale for the Assessment and Rating of Ataxia.
The prescribing information for levacetylleucine includes a warning that the drug may result in embryo-fetal harm when used during pregnancy, according to the release. The most common adverse events of levacetylleucine include abdominal pain, vomiting, difficulty swallowing and upper respiratory tract infections.
The FDA had previously granted levacetylleucine priority review, fast-track, orphan drug and rare pediatric disease designations.
Perspective
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The FDA approval of Aqneursa for adult and pediatric patients with Niemann-Pick disease type C represents a significant milestone in the treatment of this devastating disease. A pivotal, double-blinded, placebo-controlled trial has demonstrated therapeutic efficacy of this oral therapy through improvement of neurological symptoms and functional deficits, while maintaining a good safety profile.
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