Drugs / trenonacog alfa
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed | US (FDA) | — | Niemann-Pick disease type C, adult neurologic onset | 2024-03-26 | “FDA has accepted its New Drug Application (NDA) for IB1001 for the treatment of Niemann-Pick disease Type C (NPC).” intrabio.com ↗ |
Trials 6
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 23 trials · 2 met primary | ||||||
| Phase 2 | NCT03759678 | Jan 2020 → Feb 2025 | ataxia telangiectasia | IntraBio Inc | Terminated | No outcome recorded Stop: Enrollment |
| Phase 2 | NCT03759639 | Sep 2019 → Nov 2022 | Niemann-Pick disease type C | IntraBio Inc | Completed | Met primary |
| Phase 2 | NCT03759665 | Jun 2019 → Jan 2023 | Sandhoff disease, Tay-Sachs disease | IntraBio Inc | Completed | Met primary |
| Phase 2/31 trial · 1 met primary | ||||||
| Phase 2/3 | NCT00768287 | Jan 2009 → Mar 2013 | hemophilia B | Medexus Pharma, Inc. | Completed | Met primary |
| Phase 32 trials | ||||||
| Phase 3 | NCT02048111 | Mar 2014 → Oct 2015 | hemophilia B | Cangene Corporation | Withdrawn | No outcome recorded |
| Phase 3 | NCT01271868 | Nov 2010 → Dec 2016 | hemophilia B | Medexus Pharma, Inc. | Terminated | No outcome recorded |
News releases announcing trial results or a regulatory action · 17
| Date | Issuer | Release |
|---|---|---|
| 2026-01-21 | IntraBio Inc | Results IntraBio Announces Positive Pivotal Trial Results of Levacetylleucine for the Treatment of Ataxia-Telangiectasia intrabio.com ↗
IntraBio Inc. today announced positive topline results from its pivotal Phase III IB1001-303 clinical trial, “Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A Randomized, Placebo-Controlled, Double-Blind, Crossover Study” (NCT06673056) evaluating N-acetyl-L-leucine (levacetylleucine) in pediatric and adult patients with Ataxia-Telangiectasia (A-T). |
| 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. |
| 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). |
| 2018-02-25 | 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. |
| 2017-10-30 | IntraBio Inc | Regulatory INTRABIO RECEIVES EMA ORPHAN MEDICINAL DRUG DESIGNATION FOR IB1000S FOR GM2 GANGLIOSIDOSES intrabio.com ↗
IntraBio Ltd is pleased to announce that it has been granted orphan medicinal drug designation by the European Commission for its lead drug series, IB1000s, for therapeutic use in the rare lysosomal storage disorders GM2 Gangliosidoses (Sandhoff and Tay-Sachs Disease). |
All press releases naming this drug 32 releases
Evidence & citations 8 cited values
Every value below carries the sentence it was read from. 9 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | trenonacog alfa | “Trenonacog alfa (IB1001) is a recombinant factor IX (rFIX) manufactured in Chinese hamster ovary (CHO) cells.” PMID 28833808 ↗ Aug 2017 |
| Known as | IB1001 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03759678 ↗ |
| Known as | Ixinity[tm] | ChEMBL registry synonym — accepted as the source's own label CHEMBL3039538 ↗ |
| Known as | N-Acetyl-L-Leucine | “N-Acetyl-L-Leucine” NCT03759678 ↗ |
| Known as | trenacog alfa | “IB1001 trenacog alfa is an investigational recombinant factor IX (FIX) for the treatment and prevention of bleeding in individuals with haemophilia B.” PMID 22764744 ↗ Jul 2012 |
| Modality | Protein / enzyme biologic | “IB1001 is a recombinant factor IX (rFIX) manufactured in Chinese hamster ovary (CHO) cells.” PMID 28833808 ↗ Aug 2017 |
| Route | Oral | “IB1001 (N-Acetyl-L-Leucine) is a modified amino-acid ester that is orally administered.” NCT03759639 ↗ |
| Target | F9 | “Coagulation factor IX exogenous protein” CHEMBL3039538 ↗ |