Drugs / Ataluren
Regulatory milestones approvals, filings & regulatory actions · 2 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Withdrawn | EU (EMA) | Translarna | — | 2025-03-28 | europa.eu ↗ |
| Approved · conditional | EU (EMA) | Translarna | — | 2014-07-31 | europa.eu ↗ |
Trials 31 · a red edge is where a trial was stopped
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT02409004 | Feb → Mar 2015 | — | PTC Therapeutics | Completed | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT04014530 | Aug 2019 → Jun 2023 overdue | colorectal cancer, endometrial cancer | Academic Medical Center - University of Amsterdam | Recruiting | No outcome recorded |
| Phase 217 trials | ||||||
| Phase 2 | NCT04336826 | Dec 2021 → Aug 2023 | Duchenne muscular dystrophy | PTC Therapeutics | Completed | No outcome recorded |
| Phase 2 | NCT03796637 | Apr → Jun 2019 | Duchenne muscular dystrophy | PTC Therapeutics | Completed | No outcome recorded |
| Phase 2 | NCT04117880 | Dec 2018 → Jan 2021 | aniridia | PTC Therapeutics | Withdrawn | No outcome recorded |
| Phase 2 | NCT03648827 | Dec 2018 → Oct 2020 | Duchenne muscular dystrophy | PTC Therapeutics | Completed | No outcome recorded |
| Phase 2 | NCT02819557 | Jun 2016 → Feb 2018 | Duchenne muscular dystrophy | PTC Therapeutics | Completed | No outcome recorded |
| Phase 2 | NCT02647359 | Jan 2016 → Jan 2021 | aniridia | PTC Therapeutics | Completed | No outcome recorded |
| Phase 2 | NCT01141075 | Jul 2010 → Nov 2011 | inborn disorder of amino acid metabolism | PTC Therapeutics | Terminated | No outcome recorded |
| Phase 2 | NCT01009294 | Jan → Mar 2010 | Becker muscular dystrophy, Duchenne muscular dystrophy | PTC Therapeutics | Terminated | No outcome recorded |
| Phase 2 | NCT00947193 | Oct 2009 → Aug 2011 | hemophilia A, hemophilia B | PTC Therapeutics | Terminated | No outcome recorded |
| Phase 2 | NCT00847379 | Jan 2009 → May 2010 | Becker muscular dystrophy, Duchenne muscular dystrophy | PTC Therapeutics | Terminated | No outcome recorded |
| Phase 2 | NCT00759876 | Aug 2008 → May 2010 | Duchenne muscular dystrophy | PTC Therapeutics | Terminated | No outcome recorded |
| Phase 2 | NCT00592553 | Feb 2008 → Dec 2009 | Becker muscular dystrophy, Duchenne muscular dystrophy | PTC Therapeutics | Completed | No outcome recorded |
| Phase 2 | NCT00458341 | Mar 2007 → Feb 2008 | cystic fibrosis | PTC Therapeutics | Completed | No outcome recorded |
| Phase 2 | NCT00351078 | Dec 2006 → Jul 2007 | cystic fibrosis | PTC Therapeutics | Completed | No outcome recorded |
| Phase 2 | NCT00264888 | Dec 2005 → May 2007 | Duchenne muscular dystrophy | PTC Therapeutics | Completed | No outcome recorded |
| Phase 2 | NCT00237380 | Nov 2005 → May 2006 | cystic fibrosis | PTC Therapeutics | Completed | No outcome recorded |
| Phase 2 | NCT00234663 | Sep 2005 → Aug 2006 | cystic fibrosis | PTC Therapeutics | Completed | No outcome recorded |
| Phase 310 trials | ||||||
| Phase 3 | NCT03179631 | Jul 2017 → Mar 2022 | Duchenne muscular dystrophy, X-linked disease, atrophic muscular disease | PTC Therapeutics | Completed | No outcome recorded |
| Phase 3 | NCT02456103 | Aug 2015 → Jun 2017 | cystic fibrosis | PTC Therapeutics | Terminated | No outcome recorded |
| Phase 3 | NCT02139306 | Aug 2014 → Nov 2016 | cystic fibrosis | PTC Therapeutics | Completed | No outcome recorded |
| Phase 3 | NCT02107859 | May 2014 → Jun 2017 | cystic fibrosis | PTC Therapeutics | Terminated | No outcome recorded |
| Phase 3 | NCT02090959 | Mar 2014 → Jun 2018 | Duchenne muscular dystrophy, X-linked disease, atrophic muscular disease | PTC Therapeutics | Terminated | No outcome recorded |
| Phase 3 | NCT01826487 | Mar 2013 → Aug 2015 | Duchenne muscular dystrophy, X-linked disease, atrophic muscular disease | PTC Therapeutics | Completed | No outcome recorded |
| Phase 3 | NCT01557400 | May 2012 → Jan 2018 | Becker muscular dystrophy, Duchenne muscular dystrophy, neuromuscular disease caused by qualitative or quantitative defects of dystrophin | PTC Therapeutics | Completed | No outcome recorded |
| Phase 3 | NCT01247207 | Nov 2010 → Feb 2026 | Duchenne muscular dystrophy | PTC Therapeutics | Completed | No outcome recorded |
| Phase 3 | NCT01140451 | Aug 2010 → Dec 2013 | cystic fibrosis | PTC Therapeutics | Completed | No outcome recorded |
| Phase 3 | NCT00803205 | Sep 2009 → Nov 2011 | cystic fibrosis | PTC Therapeutics | Completed | No outcome recorded |
| Phase 42 trials | ||||||
| Phase 4 | NCT03256799 | Mar → Jul 2017 | cystic fibrosis | University of Alabama at Birmingham | Completed | No outcome recorded |
| Phase 4 | NCT03256968 | Jan 2017 → Dec 2018 | cystic fibrosis | University of Alabama at Birmingham | Completed | No outcome recorded |
Also used as a comparator or background therapy in 1 trial
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 2 | NCT02758626 Comparator | Nov 2016 → Feb 2021 | epilepsy | NYU Langone Health | Completed | No outcome recorded |
Evidence & citations 9 cited values
Every value below carries the sentence it was read from. 35 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | ataluren | “Ataluren (PTC124) enables ribosomal readthrough of premature stop codons, leading to production of full-length, functional proteins.” PMID 24349052 ↗ Dec 201330“Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis.” PMID 21233271 ↗ Jan 2011 |
| Known as | Ataluren(PTC124) | “Ataluren(PTC124)” NCT02409004 ↗ |
| Known as | Atalureno | ChEMBL registry synonym — accepted as the source's own label CHEMBL256997 ↗ |
| Known as | PTC124 | “Ataluren (PTC124) enables ribosomal readthrough of premature stop codons, leading to production of full-length, functional proteins.” PMID 24349052 ↗ Dec 20137“Long-Term Treatment With Ivacaftor in Combination With Ataluren (PTC124) in Subjects With Nonsense Mutation Cystic Fibrosis” NCT03256968 ↗ “Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)” NCT00759876 ↗ “Drug: Ataluren (PTC124®)” NCT02139306 ↗ “Ataluren (PTC124)” NCT03256799 ↗ “PTC124” NCT04117880 ↗ |
| Known as | Translarna | ChEMBL registry synonym — accepted as the source's own label CHEMBL256997 ↗ |
| Action | Restore | “PTC124 has been shown to partially restore CFTR production in animals with CF due to a nonsense mutation.” NCT00234663 ↗ |
| Modality | Small molecule | “a 284-Dalton 1,2,4-oxadiazole” PMID 17389552 ↗ Apr 2007 |
| Route | Oral | “A Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis” NCT00234663 ↗ |
| Target | CFTR | “PTC124 has been shown to partially restore CFTR production in animals with CF due to a nonsense mutation.” NCT00234663 ↗ |