Second Indication for Aqneursa in Ataxia Telangiectasia by Madaline Spencer| Published on: Sep 22, 2026

https://checkrare.com/second-indication-for-aqneursa-in-ataxia-telangiectasia/ The US Food and Drug Administration (FDA) has approved Aqneursa (levacetylleucine) for oral suspension to treat ataxia in patients with ataxia-telangiectasia who weigh at least 33 pounds (15 kg). It is the first treatment approved for this rare condition. Ataxia telangiectasia is a rare genetic disorder that affects the nervous system, the immune system, and many other parts of the body. The condition is typically characterized by early childhood onset of cerebellar ataxia, oculomotor apraxia, telangiectasias, choreoathetosis, a weakened immune system with frequent infections, and an increased risk of cancers such as leukemia and lymphoma. It is caused by changes in the ATM gene located on chromosome 11q22-23.