Intrabio’s Aqneursa (levacetylleucine) won FDA approval as the first treatment for ataxia-telangiectasia, following its prior U.S. authorization in Niemann-Pick disease type C. The approval expands the company’s commercially relevant footprint in rare neurodegenerative disease. Aqneursa’s entry as a first-in-indication therapy highlights the shift from supportive care to disease-modifying options in a condition with limited therapeutic history. For developers and payers, it also underscores the regulatory pathway for chemically modified amino acid approaches. Intrabio positioned the decision as a milestone for patients and care teams, with the drug now entering a new evidence and uptake phase as clinicians integrate the therapy into ataxia-telangiectasia management.