Introduction
The FDA has granted approval for Aqneursa (levacetylleucine) as the first treatment specifically aimed at addressing ataxia in adults and pediatric patients diagnosed with ataxia-telangiectasia (A-T), who weigh at least 15 kg.
Understanding Ataxia-Telangiectasia
A-T is a rare, genetic neurodegenerative condition that often begins in early childhood. The disorder is characterized by a progressive decline in muscle control and coordination, leading to difficulties with walking, balance, and speech. Unfortunately, there is currently no cure for A-T, and existing treatment options for its neurological symptoms have been scarce. Moreover, ongoing medical care must also tackle the immune deficiencies and heightened cancer risk associated with A-T, in addition to addressing the neurological issues.
About Aqneursa
Aqneursa functions as a modified amino acid, although its specific molecular action in treating A-T remains unidentified. This oral suspension is taken either two or three times a day, based on the individual’s body weight. It was previously approved in 2024 for managing neurological symptoms related to Niemann-Pick disease type C.
Clinical Trial Findings
The approval of Aqneursa for treating ataxia associated with A-T was substantiated by a randomized, double-blind, placebo-controlled phase 3 trial involving 73 participants aged between 4 and 50 years. Participants were subjected to alternating periods of treatment with Aqneursa and placebo, each lasting 12 weeks, with the order of treatment allocated randomly. Significant improvements in ataxia scores were observed during the periods when Aqneursa was administered.
Scores were assessed using the Functional Scale for Assessment and Rating of Ataxia, which measures performance in walking, standing, sitting, and speech. The scores range from 0 to 16, where lower scores indicate less impairment. The adjusted average scores revealed a significant improvement, showing 7.2 for those on Aqneursa versus 7.8 for placebo—an impressive 0.6-point difference favoring the treatment.
Adverse Reactions and Precautions
The most commonly reported adverse reactions to Aqneursa included falls, skin lacerations, and urinary tract infections. Notably, the labeling advises of potential fetal harm based on findings from animal studies. Therefore, it is imperative to rule out pregnancy prior to treatment, and individuals capable of becoming pregnant are strongly advised to utilize effective contraception during treatment and for one week following the last dose.
Community Impact
Brad Margus, founder of the A-T Children’s Project, commented, “This is a historic day for the A-T community and the patients and caregivers who have coped for decades without a treatment approved specifically for A-T. With today’s FDA decision, those impacted by A-T have a treatment option that has demonstrated benefit across a range of A-T symptoms, including those that affect daily life for patients and their loved ones.”
Disclaimer: The information provided here is for educational purposes only and should not replace professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition.
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