IntraBio Secures FDA Approval for AQNEURSA in A-T

New York, United States, September 20, 2026

IntraBio Advances Treatment for Rare Neurological Disease

The FDA approval of AQNEURSA provides a new treatment option for patients with ataxia-telangiectasia, a rare inherited disorder caused by mutations in the ATM gene. The genetic condition affects multiple body systems and is characterized by progressive neurological impairment, including cerebellar ataxia, which can interfere with walking, coordination and other motor functions. A-T can also be associated with immune-system abnormalities and increased susceptibility to certain cancers. According to IntraBio, AQNEURSA is the first treatment specifically approved by the FDA for neurological manifestations of ataxia-telangiectasia. The approval applies to adults and children aged 6 years and older. The medicine is administered orally, providing a treatment option that can be incorporated into ongoing care for eligible patients.

The FDA decision is particularly relevant for the rare-disease community because A-T affects a relatively small patient population and has historically had limited disease-specific treatment options. The approval reflects continued development of therapies designed to address neurological manifestations of rare inherited disorders.

AQNEURSA Targets Neurological Manifestations of A-T

AQNEURSA (levacetylleucine) is an oral modified amino acid developed to address neurological manifestations associated with A-T. IntraBio has investigated levacetylleucine across several rare neurological disorders characterized by impaired balance, coordination and movement. The FDA approval was supported by clinical evidence from the IB1001-301 Phase III clinical trial, a randomized, double-blind, placebo-controlled study evaluating levacetylleucine in patients with ataxia-telangiectasia. The study used the Modified International Cooperative Ataxia Rating Scale (mICARS) as a key measure of neurological function and disease severity.

The clinical development program also included an open-label extension designed to provide additional information about the treatment’s longer-term safety and tolerability. IntraBio reported that the clinical data demonstrated improvements in neurological manifestations associated with A-T, supporting the regulatory submission that led to FDA approval. As with other newly approved medicines, the FDA decision reflects an assessment of the available clinical evidence and the medicine’s benefit-risk profile. Continued post-approval monitoring will provide additional information about the long-term use of levacetylleucine in the broader patient population.

FDA Approval Marks Rare Disease Milestone

The approval of AQNEURSA represents an important development in rare neurological disease drug development, particularly for a disorder affecting children and young adults. Ataxia-telangiectasia typically begins during childhood and can progressively affect mobility, coordination and independence. The disorder can also involve complications outside the nervous system, making comprehensive patient management important. For patients and families affected by A-T, access to an FDA-approved treatment specifically indicated for neurological manifestations represents a significant addition to the therapeutic landscape. The availability of a disease-specific medicine may also contribute to greater awareness of the condition and support further clinical research into its underlying biology and potential treatment approaches.

For IntraBio, the approval marks a major regulatory achievement and expands the company’s portfolio of treatments for rare neurological diseases. The company has focused its research and development efforts on therapies for disorders involving neurological dysfunction and movement impairment, including several rare diseases. The FDA authorization of AQNEURSA (levacetylleucine) demonstrates continued progress in developing treatments for rare genetic neurological conditions. By providing an approved oral therapy for the neurological manifestations of ataxia-telangiectasia, the decision adds a new option for eligible adults and children and highlights the importance of clinical research focused on underserved rare-disease populations. The approval also underscores the broader pharmaceutical industry’s growing focus on precision treatments and rare neurological disorders, where disease-specific research can address significant unmet medical needs. As AQNEURSA becomes available to eligible patients, ongoing clinical experience and post-marketing data will contribute to understanding its role in the long-term management of A-T.

Source: IntraBio press release