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N-Acetylleucine (ACETYLLEUCINE)
N-Acetylleucine (generic name: ACETYLLEUCINE) is a acetylleucine drug. It is currently in Phase 2 development.
N-Acetylleucine works by inhibiting the enzyme leucine aminopeptidase, which breaks down certain amino acids.
N-Acetylleucine, also known as N-Acetyl-L-Leucine, is being studied as a potential treatment for several rare genetic disorders, including Ataxia-Telangiectasia, Niemann-Pick Disease, Type C, and GM2 Gangliosidosis. A pivotal study, IB1001-303, is currently investigating the effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia in a Phase III, randomized, placebo-controlled, double-blind, crossover study.
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Baseline phase 2 → approval rate
+15.3pp
Industry-wide phase 2 drugs reach approval ~15.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas).
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2031–2034 | — |
| EMA | EU | 2032–2035 | +0.7 yr |
| MHRA | GB | 2032–2035 | +0.7 yr |
| Health Canada | CA | 2032–2036 | +0.9 yr |
| TGA | AU | 2032–2036 | +1.2 yr |
| PMDA | JP | 2032–2036 | +1.5 yr |
| NMPA | CN | 2033–2037 | +2.3 yr |
| MFDS | KR | 2032–2036 | +1.4 yr |
| CDSCO | IN | 2032–2037 | +1.8 yr |
| ANVISA | BR | 2033–2037 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
At a glance
| Generic name | ACETYLLEUCINE |
|---|---|
| Drug class | acetylleucine |
| Modality | Small molecule |
| Therapeutic area | Other |
| Phase | Phase 2 |
Mechanism of action
Think of it like a traffic cop: N-Acetylleucine helps direct the flow of amino acids in the body by blocking the enzyme that breaks them down. This can help increase the levels of these amino acids, which are important for various bodily functions. By doing so, it may help alleviate certain conditions, although its exact effects are still unknown.
Approved indications
Common side effects
- Hepatitis acute
- Anticoagulation drug level above therapeutic
- Haematoma
- Inappropriate antidiuretic hormone secretion
Key clinical trials
- Pivotal Study of N-acetyl-L-leucine for CACNA1A (PHASE3)
- N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T) (PHASE2)
- 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)
- A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (PHASE3)
- N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) (PHASE2)
- N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) (PHASE2)
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 |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- N-Acetylleucine CI brief — competitive landscape report
- N-Acetylleucine updates RSS · CI watch RSS
Frequently asked questions about N-Acetylleucine
What is N-Acetylleucine?
How does N-Acetylleucine work?
What is the generic name of N-Acetylleucine?
What drug class is N-Acetylleucine in?
What development phase is N-Acetylleucine in?
What are the side effects of N-Acetylleucine?
Related
- Drug class: All acetylleucine drugs
- Therapeutic area: All drugs in Other
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing