The FDA has approved IntraBio’s AQNEURSA, or levacetylleucine, to treat ataxia in adults and pediatric patients with ataxia-telangiectasia weighing at least 15 kilograms.
AQNEURSA is the first approved treatment for ataxia-telangiectasia, or A-T, in the United States and worldwide, according to IntraBio. The medicine is commercially available, the company said.
A-T is a rare, progressive autosomal-recessive neurodegenerative disorder estimated to affect about one in 40,000 people. Progressive cerebellar degeneration can lead to worsening ataxia, affecting gait, balance, trunk control, eye movements, speech and hand coordination, alongside other systemic manifestations.
The approval is based on the pivotal IB1001-303 trial, a randomized, double-blind, placebo-controlled crossover study involving 73 patients aged 4 to 50 years with genetically confirmed A-T. The study was conducted at 10 sites in Germany, Slovakia, Spain, Switzerland, the United Kingdom and the United States.
AQNEURSA met its primary efficacy endpoint on the Scale for the Assessment and Rating of Ataxia, or SARA. The treatment showed a 1.9-point improvement versus placebo, with a 95% confidence interval of negative 2.7 to negative 1.1 and a two-sided p-value below 0.001.
FDA’s primary outcome assessment used the functional SARA, a modified measure covering gait, sitting, stance and speech disturbance. Patients who received AQNEURSA had a mean treatment difference of negative 0.6 points versus placebo, with a 95% confidence interval of negative 0.9 to negative 0.2 and a p-value below 0.001.
Seventy patients, or 96%, completed the trial and received both placebo and AQNEURSA. The company said results were consistent across pediatric and adult subgroups.
No treatment-related serious adverse events, deaths or treatment-related discontinuations occurred. The most common adverse reactions occurring in at least 5% of patients and more often than placebo were falls and skin lacerations.
AQNEURSA was previously approved in September 2024 for neurological manifestations of Niemann-Pick disease type C in adults and pediatric patients weighing at least 15 kilograms.
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