Treats neurological manifestations of Niemann-Pick disease type C (NPC). Sold as Aqneursa. Every FDA application for this active ingredient.
As of Nov 14, 2026, FDA lists 1 application for Levacetylleucine (sold as Aqneursa), first approved Sep 24, 2024, with 1 new use approved and 0 published complete response letters.
| Product | Part | Spending, 2025 | Change | Beneficiaries |
|---|---|---|---|---|
| Aqneursa | Part D | $11.6M | New | 27 |
| Study | Phase | Status | Posted |
|---|---|---|---|
| A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)Ataxia-Telangiectasia, Ataxia-Telangiectasia (A-T) · IntraBio Inc | Phase 3 | Active, not recruiting | Nov 4, 2024 |
Applications, approvals and products come from FDA's Drugs@FDA data files, refreshed weekly, grouped by active ingredient as FDA spells it. Complete response letters are FDA's published letters. Medicare spending is CMS's Part D and Part B dashboard, matched to this ingredient by generic name; shortages are FDA's shortage list, matched by application number; clinical trials are ClinicalTrials.gov studies naming the ingredient as an intervention, refreshed daily; safety programs are FDA's REMS list, matched by application number or, for shared programs, by ingredient. Drugs@FDA
