Aqneursa cleared for ataxia in ataxia-telangiectasia
- Sponsor
- IntraBio Inc.
- Drug
- Aqneursa · levacetylleucine
- Indication
- ataxia in adults and pediatric patients with ataxia-telangiectasia weighing at least 15 kg (label expansion; originally approved 2024 for neurological manifestations of Niemann-Pick disease type C)
FDA approved a supplemental NDA for IntraBio's Aqneursa (levacetylleucine) for ataxia in adults and pediatric patients with ataxia-telangiectasia weighing at least 15 kg.
What this means
Ataxia-telangiectasia (A-T) is a rare inherited neurodegenerative disease caused by mutations in the ATM gene. Children with A-T progressively lose muscle control and coordination (ataxia) starting in early childhood, and the disease also causes small dilated blood vessels (telangiectasias), immune deficiencies, and an elevated risk of cancer. There is no cure, and until now treatment options specifically targeting the ataxia component were limited. Aqneursa (levacetylleucine) is IntraBio's oral suspension of a modified form of the amino acid leucine. FDA first approved Aqneursa in 2024 for neurological manifestations of Niemann-Pick disease type C, and on September 19, 2026 — the PDUFA target action date — the FDA approved a supplemental application that extends the label to treat ataxia in adults and pediatric patients with A-T who weigh at least 15 kg (about 33 pounds). Aqneursa becomes the first FDA-approved treatment for ataxia in patients with A-T. Approval was supported by a randomized, double-blind, placebo-controlled Phase 3 crossover study of 73 A-T patients ages 4-50 (26 adults, 47 pediatric; 96% study completion). Patients scored significantly better on the functional Scale for Assessment and Rating of Ataxia (fSARA) while taking Aqneursa than on placebo. The indication received Orphan Drug designation and Priority Review. Common adverse events were fall, skin laceration, and urinary tract infection; simultaneous use with N-acetyl-DL-leucine should be avoided.
From the source
The U.S. Food and Drug Administration has approved Aqneursa (levacetylleucine) for oral suspension to treat ataxia in adults and pediatric patients with ataxia-telangiectasia weighing at least 15 kg (about 33 pounds). Aqneursa was previously approved in 2024 to treat the neurological manifestations of Niemann-Pick disease type C. Today’s approval makes Aqneursa the first treatment approved for ataxia in patients with ataxia-telangiectasia.
Condition
Ataxia-telangiectasia is a rare, inherited neurodegenerative disorder caused by mutations in the ATM gene. It primarily affects the nervous system, causing progressive loss of muscle control and coordination (ataxia) that typically begins in early childhood. The disease also causes small dilated blood vessels (telangiectasias), immune deficiencies, and an elevated risk of cancer. There is no cure for ataxia-telangiectasia, and treatment options for the neurological symptoms of the disease have been limited.
Data Supporting Aqneursa
The effectiveness of Aqneursa was evaluated in a randomized, double-blind, placebo-controlled, two-period crossover study (NCT06673056) of 73 patients aged 4 years or older with a confirmed diagnosis of ataxia-telangiectasia. Patients were randomly assigned to receive Aqneursa followed by placebo, or placebo followed by Aqneursa, with each treatment period lasting 12 weeks. Of the 73 patients (26 adults and 47 pediatric patients), 38 were female and 35 were male. The median age at treatment initiation was 13 years (range: 4 to 50 years). A total of 70 patients (96%) completed the study.
Efficacy was assessed using the functional Scale for Assessment and Rating of Ataxia (fSARA), a modified clinical tool evaluating gait, sitting, stance, and speech disturbance, with scores ranging from 0 (best neurological status) to 16 (worst). When patients were taking Aqneursa, they scored better on the fSARA compared to when these patients were taking placebo and showed significant improvement in neurological function.
Safety Information
There are no contraindications for Aqneursa, although this drug may cause fetal harm based on data from animal studies. The most common adverse reactions in patients with ataxia-telangiectasia were fall, skin laceration, and urinary tract infection.
Aqneursa interacts with N-acetyl-DL-leucine and simultaneous use should be avoided. Patients receiving P-glycoprotein (P-gp) substrates should be monitored more frequently for related adverse reactions when used with Aqneursa.
Designation
Aqneursa received Orphan Drug designation and Priority Review for the ataxia-telangiectasia indication. Approval was granted to IntraBio Inc.
Reproduced from FDA Drugs (Whats New) · view original · public domain or open-licensed government work
Drug Watch — recent news on Aqneursa
- FDA Approves Aqneursa for Ataxia-Telangiectasia - Medical Dialogues
- IntraBio Announces U.S. FDA Approval of AQNEURSA® (levacetylleucine) for Ataxia-Telangiectasia - The National Law Review
- IntraBio's Aqneursa scores expansion, now the first treatment for rare ataxia - Fierce Pharma
- FDA Approves IntraBio’s Aqneursa as First Treatment for Ataxia-Telangiectasia - contractpharma.com
- Intrabio’s Aqneursa wins FDA nod as first ataxia-telangiectasia drug - bioworld.com