On September 21, 2026, the FDA approved IntraBio Inc.'s AQNEURSA (levacetylleucine) as the first and only treatment for ataxia in patients with ataxia-telangiectasia (A-T) weighing 15 kg or more. This marks the second FDA approval for AQNEURSA, which was approved in September 2024 for Niemann-Pick disease type C. The approval is supported by a Phase 3 trial of 73 patients published in The Lancet Neurology, which demonstrated significant improvements in motor function and ataxia symptoms.
AQNEURSA FDA approval for ataxia-telangiectasia
- ▪IntraBio Inc. announced that AQNEURSA is commercially available in the United States immediately following its approval on September 21, 2026.
- ▪The U.S. Food and Drug Administration approved IntraBio Inc.'s AQNEURSA (levacetylleucine) on September 21, 2026, for the treatment of ataxia in adult and pediatric patients with ataxia-telangiectasia weighing 15 kg or more.
- ▪AQNEURSA is the first and only drug approved for the treatment of ataxia-telangiectasia anywhere in the world.
Ataxia-telangiectasia disease characteristics
- ▪Ataxia-telangiectasia affects an estimated 1 in 40,000 people and is characterized by progressive cerebellar degeneration resulting in worsening ataxia that affects gait, balance, speech, and coordination.
- ▪Ataxia-telangiectasia is a rare, progressive, and prematurely fatal autosomal-recessive neurodegenerative disorder caused by biallelic pathogenic variants in the ATM gene.
Phase 3 trial results
- ▪In the IB1001-303 trial, AQNEURSA demonstrated statistically significant improvement compared to placebo on the Scale for the Assessment and Rating of Ataxia, with a treatment difference of -1.9 points.
- ▪The FDA approval of AQNEURSA was supported by the IB1001-303 study, a randomized, double-blind, placebo-controlled, crossover trial enrolling 73 patients aged 4 to 50 years with genetically confirmed ataxia-telangiectasia.
- ▪The IB1001-303 trial results, published in the July 2026 issue of The Lancet Neurology, showed that 70 patients completed the study with no treatment-related serious adverse events or deaths.
- ▪In the IB1001-303 trial, patients receiving AQNEURSA showed greater improvement in the functional Scale for the Assessment and Rating of Ataxia score, with a mean treatment difference of -0.6.
AQNEURSA mechanism of action
- ▪Preclinical studies of AQNEURSA showed it normalized lysosomal function, reduced substrate accumulation, improved mitochondrial ATP production, and reduced neuroinflammation.
- ▪The proposed mechanism of action for AQNEURSA is the normalization of glucose metabolism, which is correlated with enhanced cerebellar activity, though its distinct molecular target is unknown.
Prior Niemann-Pick disease approval
- ▪AQNEURSA is indicated for Niemann-Pick disease type C in adult and pediatric patients weighing 15 kg or more, taken orally as a suspension.
- ▪The September 21, 2026 approval marks the second FDA-approved indication for AQNEURSA, following its initial approval in September 2024 for treating neurological manifestations of Niemann-Pick disease type C.
Patient community response
- ▪IntraBio Inc. offers the AQNEURSA Cares support program to provide financial assistance and educational resources to help eligible patients navigate insurance coverage and access the treatment.
- ▪Brad Margus, founder of the A-T Children's Project, described the FDA approval of AQNEURSA as a historic day for the ataxia-telangiectasia community who have coped for decades without an approved treatment.
Story comments
Loading comments…