Drugs / Levacetylleucine
Levacetylleucine
also known as
N-acetyl-l-leucine · Acetyl-l-leucine · Aqneursa · IB1001 · Levacetylleucina · N-acetyl leucine · NALL · NSC-206316
Regulatory milestones approvals, filings & regulatory actions · 2 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed Niemann-Pick disease type C, adult neurologic onset 1 milestone | |||||
| Filed | US (FDA) | — | Niemann-Pick disease type C, adult neurologic onset | 2024-03-26 | “Administration (FDA) has accepted its New Drug Application (NDA) for IB1001 for the treatment of Niemann-Pick disease type C (NPC).” intrabio.com ↗ |
| Approved Indication not stated 1 milestone | |||||
| Approved | US (FDA) | AQNEURSA | — | 2024-09-24 | fda.gov ↗ |
Trials 4
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 33 trials · 2 met primary | ||||||
| Phase 3 | NCT07221292 | Sep 2026 → Aug 2027 expected | episodic ataxia type 2, migraine, familial hemiplegic, 1, spinocerebellar ataxia type 6 | IntraBio Inc | Not yet recruiting | No outcome recorded |
| Phase 3 | NCT06673056 | Mar 2025 → Dec 2027 expected | ataxia telangiectasia | IntraBio Inc | Active not recruiting | Met primary |
| Phase 3 | NCT05163288 | Jun 2022 → Jun 2030 expected | Niemann-Pick disease type C | IntraBio Inc | Recruiting | Met primary |
| Phase not stated1 trial | ||||||
| — | NCT07380165 | — | ataxia telangiectasia | IntraBio Inc | Available | No outcome recorded |
News releases announcing trial results or a regulatory action · 20
| Date | Issuer | Release |
|---|---|---|
| 2026-01-21 | IntraBio Inc | Regulatory IntraBio Receives European Commission Approval of AQNEURSA® for the Treatment of Niemann-Pick Type C Disease intrabio.com ↗
IntraBio Inc. today announced that the European Commission granted marketing authorization to AQNEURSA ® (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick Type C (NPC) disease, following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). |
| 2026-01-21 | IntraBio Inc | Results IntraBio Announces Positive Pivotal Trial Results of Levacetylleucine for the Treatment of Ataxia-Telangiectasia intrabio.com ↗
Treatment with levacetylleucine demonstrated a statistically significant and clinically meaningful -1.88 point improvement of the SARA score compared to placebo (-1.92 on levacetylleucine vs -0.14 on placebo, p<0.001). |
| 2024-09-25 | IntraBio Inc | Regulatory IntraBio Announces U.S. FDA Approval of AQNEURSA for the Treatment of Niemann-Pick Disease Type C intrabio.com ↗
IntraBio Inc., a leader in the discovery and development of innovative drugs for rare neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved AQNEURSA (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥ 15 kg. |
| 2024-03-26 | IntraBio Inc | Regulatory US FDA Acceptance of NDA for IB1001 for NPC intrabio.com ↗
IntraBio Inc today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for IB1001 for the treatment of Niemann-Pick disease Type C (NPC). |
| 2023-08-08 | IntraBio Inc | Results Positive Pivotal Trial Results of IB1001 for the Treatment of Niemann-Pick Disease Type C intrabio.com ↗
IntraBio Inc today announced positive topline results from the pivotal, Phase III IB1001-301 clinical trial (NCT05163288), showing IB1001 significantly improved symptoms, functioning, quality of life, and cognition in pediatric and adult patients with Niemann-Pick disease Type C (NPC). |
| 2021-09-14 | IntraBio Inc | Results INTRABIO REPORTS FURTHER DETAIL ON POSITIVE DATA FROM IB1001 MULTINATIONAL CLINICAL TRIAL FOR THE TREATMENT OF GM2 GANGLIOSIDOSIS (TAY-SACHS AND SANDHOFF DISEASE) intrabio.com ↗
IntraBio Inc announced positive results from the full data set for its multinational clinical trial of IB1001 (N-acetyl-L-leucine) for the treatment of GM2 Gangliosidosis (“GM2”; Tay-Sachs and Sandhoff disease). |
| 2021-08-19 | IntraBio Inc | Results IntraBio Reports Statistically Significant and Clinically Meaningful Improvements in the Use of IB1001 For Treatment of GM2 Gangliosidosis intrabio.com ↗
IntraBio Inc announced positive data from its multinational clinical trial of IB1001 for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease). |
| 2020-10-19 | IntraBio Inc | Results IntraBio Reports Further Detail on Positive Data from IB1001 Multinational Clinical Trial for the Treatment of Niemann-Pick disease Type C intrabio.com ↗
Oxford, U.K. — IntraBio Inc today announced positive results from the full data set for its multinational clinical trial of IB1001 (N-acetyl-L-leucine) for the treatment of Niemann-Pick disease Type C (NPC). |
| 2020-09-30 | IntraBio Inc | Results IntraBio Reports Positive Data from IB1001 Multinational Clinical Trial for the Treatment of Niemann-Pick disease Type C intrabio.com ↗
IB1001 demonstrated a statistically significant and clinically meaningful improvement in symptoms, functioning, and quality of life in both primary and topline secondary endpoints for both pediatric and adult patients with NPC. |
| 2020-03-25 | IntraBio Inc | Regulatory IntraBio Fast Track Designation for Niemman-Pick Disease type C intrabio.com ↗
IntraBio Inc. is pleased to share that the US Food and Drug Administration (FDA) has granted Fast Track designation to its lead compound (IB1001) for the treatment of Niemann-Pick disease Type C (NPC). |
| 2020-03-25 | IntraBio Inc | Regulatory IntraBio Fast Track Designation for GM2 Gangliosidosis intrabio.com ↗
IntraBio Inc. is pleased to share that the US Food and Drug Administration (FDA) has granted Fast Track designation to its lead compound (IB1001) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease). |
| 2020-03-09 | IntraBio Inc | Regulatory INTRABIO NEUROPROTECTION AND DISEASE MODIFICATION FOR GM2 GANGLIOSIDOSIS intrabio.com ↗
The acceptance of the Extension Phases provides US patients enrolled in the current trial the opportunity to continue treatment with IB1001 if the principal investigator determines it is in the patient’s best interest. |
| 2020-01-17 | IntraBio Inc | Regulatory IntraBio Neuroprotection and Disease Modification for Niemann-Pick C Extension Phase Approved in Europe intrabio.com ↗
IntraBio Inc is pleased to share that the Extension Phase for the IB1001-201 Clinical Trial has been approved in all European countries where the trial is being conducted. |
| 2020-01-17 | IntraBio Inc | Results IntraBio Announces Clinical Results for Treatment of Dementia intrabio.com ↗
Recent compassionate-use studies with IB1000s in patients with Lewy Body Dementia and Fronto-Temporal Dementia showed meaningful improvement in quality of life, cognition, mobility, speech, and a disease-modifying effect. |
| 2019-02-14 | IntraBio Inc | Regulatory IntraBio Investigational New Drug Application Approved by the FDA for the Treatment of Niemann-Pick Disease Type C intrabio.com ↗ |
| 2019-02-11 | IntraBio Inc | Regulatory IntraBio Investigational New Drug Application Approved by the FDA for the Treatment of Tay-Sachs and Sandhoff Disease intrabio.com ↗
The IND approval of IB1001-202 allows IntraBio to move forward with the trial at U.S. clinical sites. |
| 2018-10-01 | IntraBio Inc | Regulatory INTRABIO RECEIVES FDA RARE PEDIATRIC DISEASE DESIGNATION FOR IB1000S FOR GM2 GANGLIOSIDOSES intrabio.com ↗
IntraBio Inc is pleased to announce that the US Food and Drug Administration has granted a Rare Pediatric Disease Designation for their lead compound series, IB1000s, for the treatment of GM2 Gangliosidoses (Tay-Sachs and Sandhoff diseases). |
| 2018-08-01 | IntraBio Inc | Regulatory INTRABIO RECEIVES FDA & EMA ORPHAN DRUG DESIGNATIONS FOR IB1000S FOR SPINOCEREBELLAR ATAXIAS intrabio.com ↗ |
| 2018-04-12 | IntraBio Inc | Regulatory INTRABIO RECEIVES FDA RARE PEDIATRIC DISEASE DESIGNATION FOR IB1000S FOR NIEMANN-PICK DISEASE TYPE C intrabio.com ↗
IntraBio Inc is pleased to announce that the US Food and Drug Administration has granted a Rare Pediatric Disease Designation for their lead compound series, IB1000s, for the treatment of Niemann-Pick disease type C. |
| 2018-02-25 | IntraBio Inc | Regulatory INTRABIO RECEIVES FDA ORPHAN DRUG DESIGNATION FOR IB1000S FOR NIEMANN-PICK DISEASE TYPE C intrabio.com ↗
IntraBio Inc has announced that it has been granted orphan designation by the US Food and Drug Administration for its lead drug series, IB1000s, for therapeutic use in the rare lysosomal storage disorders Niemann-Pick disease type C. |
All press releases naming this drug 35 releases
Evidence & citations 11 cited values
Every value below carries the sentence it was read from. 7 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Levacetylleucine | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07380165 ↗ |
| Known as | Acetyl-l-leucine | ChEMBL registry synonym — accepted as the source's own label CHEMBL56021 ↗ |
| Known as | Aqneursa | ChEMBL registry synonym — accepted as the source's own label CHEMBL56021 ↗ |
| Known as | IB1001 | “The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.” NCT07221292 ↗ |
| Known as | Levacetylleucina | ChEMBL registry synonym — accepted as the source's own label CHEMBL56021 ↗ |
| Known as | N-acetyl leucine | ChEMBL registry synonym — accepted as the source's own label CHEMBL56021 ↗ |
| Known as | N-acetyl-l-leucine | “We evaluated the safety and efficacy of N-acetyl-l-leucine (NALL), an agent that potentially ameliorates lysosomal and metabolic dysfunction, for the treatment of Niemann-Pick...” PMID 38294974 ↗ Feb 2024 |
| Known as | NALL | “We evaluated the safety and efficacy of N-acetyl-l-leucine (NALL), an agent that potentially ameliorates lysosomal and metabolic dysfunction, for the treatment of Niemann-Pick...” PMID 38294974 ↗ Feb 2024 |
| Known as | NSC-206316 | ChEMBL registry synonym — accepted as the source's own label CHEMBL56021 ↗ |
| Modality | Small molecule | “N-Acetyl-L-Leucine is a modified amino-acid ester” NCT05163288 ↗ |
| Route | Oral | “orally administered levacetylleucine” PMID 42309084 ↗ Jul 2026 |