Drugs / Drisapersen
Regulatory milestones approvals, filings & regulatory actions · 4 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Rejected (CRL) | US (FDA) | Kyndrisa | DMD-related muscular dystrophy | 2016-01-14 | “FDA issued a Complete Response letter to the Company's New Drug Application (NDA) for Kyndrisa TM (drisapersen) for the treatment of Duchenne muscular dystrophy” biomarin.com ↗ |
| Filed | US (FDA) | — | DMD-related muscular dystrophy | 2015-06-29 | “today announced the U.S. Food and Drug Administration (FDA) has accepted for review the submission of a New Drug Application (NDA) for drisapersen” biomarin.com ↗ “the U.S. Food and Drug Administration (FDA) had accepted for review the submission of a New Drug Application (NDA) for drisapersen” biomarin.com ↗ |
| Filed | EU (EMA) | — | DMD-related muscular dystrophy | 2015-06-08 | “today announced the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for drisapersen for the treatment of Duchenne Muscular Dystrophy” biomarin.com ↗ “the European Medicines Agency (EMA) had accepted for review the submission of a Marketing Authorization Application (MAA) for drisapersen” biomarin.com ↗ “announced today the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for drisapersen” biomarin.com ↗ |
| Filed | US (FDA) | — | DMD-related muscular dystrophy | 2015-04-27 | “announced completion of the rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for drisapersen” biomarin.com ↗ |
Trials 9
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT01128855 | Jul 2010 → Oct 2011 | Duchenne muscular dystrophy | GlaxoSmithKline | Completed | No outcome recorded |
| Phase 23 trials · 1 met primary · 1 missed | ||||||
| Phase 2 | NCT01462292 | Oct 2011 → May 2013 | muscular dystrophy | GlaxoSmithKline | Completed | Missed primary |
| Phase 2 | NCT01153932 | Sep 2010 → Mar 2012 | muscular dystrophy | GlaxoSmithKline | Completed | Met primary |
| Phase 2 | NCT01910649 | Mar 2008 → Sep 2016 | muscular dystrophy | BioMarin Pharmaceutical | Terminated | No outcome recorded |
| Phase 34 trials · 1 missed | ||||||
| Phase 3 | NCT01890798 | Jan 2014 | muscular dystrophy | GlaxoSmithKline | Withdrawn | No outcome recorded Stop: Enrollment |
| Phase 3 | NCT01803412 | May 2013 → Jun 2016 | muscular dystrophy | BioMarin Pharmaceutical | Terminated | No outcome recorded |
| Phase 3 | NCT01480245 | Sep 2011 → Mar 2014 | muscular dystrophy | GlaxoSmithKline | Terminated | No outcome recorded |
| Phase 3 | NCT01254019 | Dec 2010 → Jun 2013 | Duchenne muscular dystrophy | GlaxoSmithKline | Completed | Missed primary |
| Phase not stated1 trial | ||||||
| — | NCT02636686 | — | Duchenne muscular dystrophy | BioMarin Pharmaceutical | No longer available | No outcome recorded |
News releases announcing trial results or a regulatory action · 13
| Date | Issuer | Release |
|---|---|---|
| 2016-05-31 | BioMarin Pharmaceutical | Regulatory BioMarin Announces Withdrawal of Market Authorization Application for Kyndrisa™ (drisapersen) in Europe biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that it has withdrawn its Kyndrisa™ (drisapersen) Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) following discussions at the May 2016 Committee for Medicinal Products for Human Use (CHMP) meeting. |
| 2016-01-14 | BioMarin Pharmaceutical | Regulatory FDA Issues Complete Response Letter for KyndrisaTM for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping biomarin.com ↗ |
| 2015-12-18 | BioMarin Pharmaceutical | Regulatory BioMarin Announces That FDA Has Advised it Will Not Take Action on the Kyndrisa™ (drisapersen) New Drug Application by the PDUFA Date biomarin.com ↗ |
| 2015-11-20 | BioMarin Pharmaceutical | Results BioMarin Announces Data Analysis Demonstrating Consistent Efficacy of Kyndrisa™ (drisapersen) in Comparable Patients Across Three Randomized Studies biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced data analysis for Kyndrisa demonstrating consistent evidence of efficacy in comparable patients across three randomized, placebo-controlled studies that were conducted contemporaneously. |
| 2015-10-15 | BioMarin Pharmaceutical | Regulatory BioMarin Announces FDA Advisory Committee to Review Drisapersen for Treatment of Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) will review the New Drug Application (NDA) for drisapersen. |
| 2015-08-19 | BioMarin Pharmaceutical | Regulatory BioMarin Receives Rare Pediatric Disease Designation From FDA for Drisapersen for the Potential Treatment of Duchenne Muscular Dystrophy biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation for drisapersen, a potential treatment for patients with Duchenne Muscular Dystrophy (DMD) who are amenable to exon 51 skipping treatment. |
| 2015-06-29 | BioMarin Pharmaceutical | Regulatory BioMarin Announces FDA Accepts Drisapersen NDA for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping biomarin.com ↗
The FDA has granted drisapersen Priority Review status, which is designated to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists. |
| 2015-06-25 | BioMarin Pharmaceutical | Regulatory BioMarin Announces EMA Validates MAA for Drisapersen for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for drisapersen for the treatment of Duchenne Muscular Dystrophy amenable to exon 51 skipping. |
| 2015-06-08 | BioMarin Pharmaceutical | Regulatory BioMarin Submits Drisapersen MAA to EMA for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping biomarin.com ↗ |
| 2015-04-30 | BioMarin Pharmaceutical | Regulatory BioMarin Announces First Quarter 2015 Financial Results and Company Update biomarin.com ↗
Rolling NDA Submission Completed for Drisapersen |
| 2015-04-27 | BioMarin Pharmaceutical | Regulatory BioMarin Completes Rolling NDA Submission to FDA for Drisapersen for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping biomarin.com ↗ |
| 2013-09-20 | GlaxoSmithKline | Results GSK and Prosensa announce primary endpoint not met in Phase III study of drisapersen in patients with Duchenne Muscular Dystrophy gsk.com ↗
The difference in 6MWD (mean (CI) 10.33m (-14.65, 35.31), p=0.415) between drisapersen and placebo groups did not reach statistical significance. |
| 2013-06-27 | GlaxoSmithKline | Regulatory GlaxoSmithKline’s drisapersen (previously GSK2402968/PRO051) to receive Food and Drug Administration Breakthrough Therapy designation gsk.com ↗
GlaxoSmithKline plc (GSK) received verbal notification yesterday that its investigational compound drisapersen (previously GSK2402968/PRO051) has been granted Breakthrough Therapy designation by the United States Food and Drug Administration (FDA) for the potential treatment of patients with Duchenne Muscular Dystrophy. |
All press releases naming this drug 17 releases
Evidence & citations 9 cited values
Every value below carries the sentence it was read from. 12 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Drisapersen | ClinicalTrials.gov intervention name — accepted as the source's own label NCT01890798 ↗ |
| Known as | GSK-2402968A | ChEMBL registry synonym — accepted as the source's own label CHEMBL2108735 ↗ |
| Known as | GSK2402968 | “This 188-week open-label extension of the dose-escalation study assessed the long-term efficacy, safety, and pharmacokinetics of drisapersen (PRO051/GSK2402968), 6 mg/kg...” PMID 27588424 ↗ Sep 2016 |
| Known as | H51AON23 | ChEMBL registry synonym — accepted as the source's own label CHEMBL2108735 ↗ |
| Known as | PRO-051 | ChEMBL registry synonym — accepted as the source's own label CHEMBL2108735 ↗ |
| Action | Restore | “Antisense-oligonucleotide-induced exon skipping allows synthesis of partially functional dystrophin.” PMID 25209738 ↗ Sep 2014 |
| Modality | Antisense oligonucleotide | “a 2'-O-methyl-phosphorothioate antisense oligonucleotide” PMID 25209738 ↗ Sep 2014 |
| Route | Subcutaneous | “Weekly subcutaneous injection” NCT01128855 ↗ |
| Target | DMD | “dystrophin mutations correctable by drisapersen-induced DMD Exon 51 skipping” NCT01890798 ↗ |