Cindy Howry: FDA Approves First Treatment for Ataxia-Telangiectasia
Cindy Howry, Executive Principal at Howry Solutions, shared a post on LinkedIn:
”FDA Approves First Treatment for Ataxia-Telangiectasia
The FDA has approved Aqneursa (levacetylleucine), an oral suspension that marks the first-ever treatment specifically approved for ataxia—a progressive neurological condition that affects movement and coordination—in patients with ataxia-telangiectasia.
The approval applies to adults and children weighing at least 15 kg (about 33 pounds).
This represents a significant milestone for a rare genetic disorder that has had no targeted treatment options until now.
Aqneursa builds on the medication’s prior FDA approval in 2024 for treating neurological symptoms in another rare genetic condition, Niemann-Pick disease type C.”
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