Last reviewed · How we verify
Aqneursa (LEVACETYLLEUCINE)
Aqneursa works by modifying the activity of a specific enzyme involved in the breakdown of fatty substances in the body.
At a glance
| Generic name | LEVACETYLLEUCINE |
|---|---|
| Sponsor | Intrabio |
| Modality | Small molecule |
| Therapeutic area | Metabolic |
| Phase | FDA-approved |
| First approval | 2024 |
Mechanism of action
The distinct molecular target for levacetylleucine in the treatment of NPC is unknown.
Approved indications
- Niemann-Pick disease type C (NPC)
Common side effects
- Upper respiratory tract infection
- Abdominal pain
- Dysphagia
- Vomiting
- Thrombocytopenia
Drug interactions
- N-acetyl-DL-leucine
- N-acetyl-D-leucine
- P-glycoprotein (P-gp) Transporter Substrates
Key clinical trials
- A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (PHASE3)
- Expanded Access Program (EAP) of Levacetylleucine for Ataxia-Telangiectasia (A-T)
- A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T) (PHASE3)
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |