FDA approves Aqneursa as first treatment for ataxia in ataxia-telangiectasia
The FDA has approved Aqneursa as the first treatment specifically indicated for ataxia in adults and children with ataxia-telangiectasia weighing at least 15kg.
Aqneursa, or levacetylleucine, is approved to treat the neurological manifestation of ataxia in patients with the rare inherited disorder ataxia-telangiectasia.
The approval introduces the first FDA-authorised therapy specifically targeting ataxia in this patient population, addressing an area that previously lacked an approved treatment.
Field | Content |
Alert Type | Approval |
Drug Name | Levacetylleucine |
Brand Name | Aqneursa |
Regulatory Authority | U.S. Food and Drug Administration |
Approval Type | New drug approval |
Indication | Treatment of ataxia in adults and paediatric patients with ataxia-telangiectasia weighing at least 15kg |
Therapy Area(s) | Rare disease; neurology; genetic disease |
Technology or Modality | Small-molecule therapy |
Geography | United States |
What Happened | The FDA approved Aqneursa (levacetylleucine) for treatment of ataxia in adults and children with ataxia-telangiectasia weighing at least 15kg. |
Why It Matters | Aqneursa is the first FDA-approved treatment specifically addressing the neurological symptom of ataxia in patients with this rare genetic disorder. |
Supporting Context | Ataxia-telangiectasia is an inherited multisystem disorder characterised by progressive neurological impairment, including worsening coordination and balance. |
Key Takeaway | Aqneursa introduces the first FDA-approved therapy specifically for ataxia associated with ataxia-telangiectasia. |
What to Watch | Clinical uptake, access for eligible paediatric and adult patients and longer-term evidence on neurological outcomes. |
Primary Source | U.S. Food and Drug Administration |
Relevant Date | 18 September 2026 |
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