FDA approves levacetylleucine for the treatment of Niemann-Pick disease, type C

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The FDA has recently approved levacetylleucine (Aqneursa; IntraBio) for the treatment of neurological symptoms associated with Niemann-Pick disease, type C (NPC), in adults and pediatric patients weighing a minimum of 15 kilograms.¹

NPC is a rare genetic disease that causes neurological symptoms and organ dysfunction.This rare disease is caused by changes in either the NPC1 or NPC2 gene, affecting the transportation of cholesterol and other lipids within a cell, resulting in organ damage. According to the FDA, individuals with NPC live for approximately 13 years on average.¹

This approval comes on the heels of another recent FDA approval for NPC, arimoclomol (Miplyffa; Zevra Therapeutics), making it the first FDA-approved drug to treat NPC in adults and children aged 2 years and older.² According to the federal agency, this approval is for arimoclomol in combination with the enzyme inhibitor miglustat to treat NPC that results in progressive neurological symptoms and organ dysfunction.

Read more: FDA approves arimoclomol as first drug to treat Neimann-Pick disease, type C

The levacetylleucine application was supported by various FDA designations including Priority Review, Fast Track, Orphan Drug and Rare Pediatric Disease designations.¹

“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,” said Janet Maynard, MD, MHS, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, in the FDA’s Center for Drug Evaluation and Research. “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.”¹

The safety and efficacy of levacetylleucine were assessed in a randomized, double-blind, placebo-controlled, 2-period, 24-week crossover study. Each treatment period lasted 12 weeks, and 60 patients participated.¹

To qualify, patients had to be 4 years or older, have a confirmed diagnosis of NPC, and exhibit at least mild neurological symptoms related to the disease. Participants were allowed to use miglustat, an enzyme inhibitor, as part of their background treatment during the study.¹

The primary efficacy endpoint was a modified version of the Scale for the Assessment and Rating of Ataxia (SARA), known as the functional SARA (fSARA). This version focuses on the gait, sitting, stance, and speech disturbance sections of the original SARA, with adjusted scoring. On average, participants treated with levacetylleucine for 12 weeks had better fSARA scores compared to when they received a placebo.¹

The prescribing information includes a warning that levacetylleucine may cause harm to an embryo or fetus if used during pregnancy. Females should notify their health care provider if they are pregnant or suspect pregnancy before starting levacetylleucine.¹

Common side effects include abdominal pain, difficulty swallowing, upper respiratory tract infections, and vomiting.¹

Levacetylleucine is taken orally up to 3 times daily, with or without food. The recommended dosage is based on the patient's body weight, as specified in the prescribing information.¹

References:

1. FDA. FDA Approves New Drug to Treat Niemann-Pick Disease, Type C. US Food and Drug Administration. September 24, 2024. Accessed September 25, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-new-drug-treat-niemann-pick-disease-type-c?utm_medium=email&utm_source=govdelivery

2. FDA approves first treatment for Niemann-Pick Disease, Type C. FDA. Press release. September 20, 2024. Accessed September 25, 2024. https://content.govdelivery.com/accounts/USFDA/bulletins/3b708bf