Drugs / Eteplirsen
Regulatory milestones approvals, filings & regulatory actions · 8 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed DMD-related muscular dystrophy 6 milestones | |||||
| Refused (EMA) | EU (EMA) | — | DMD-related muscular dystrophy | 2018-09-21 | “The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) confirmed its May 31, 2018 negative opinion for a Conditional Marketing...” sarepta.com ↗ “CHMP of the European Medicines Agency (EMA), has confirmed its 31 May 2018 negative opinion for a Conditional Marketing Application for eteplirsen.” sarepta.com ↗ |
| Refused (EMA) | EU (EMA) | EXONDYS | DMD-related muscular dystrophy | 2018-06-01 | “announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), has adopted a negative opinion for EXONDYS® (eteplirsen)” sarepta.com ↗ |
| Filed | EU (EMA) | — | DMD-related muscular dystrophy | 2016-12-19 | “EMA validated the previously submitted Marketing Authorization application (MAA) for eteplirsen to treat Duchenne muscular dystrophy” sarepta.com ↗ |
| Filed | US (FDA) | — | DMD-related muscular dystrophy | 2016-02-25 | “2015 marked a year of significant achievements for Sarepta, the most important being the filing of our New Drug Application for eteplirsen” sarepta.com ↗ |
| Filed | US (FDA) | — | DMD-related muscular dystrophy | 2015-08-25 | “the New Drug Application (NDA) that Sarepta submitted to the U.S. Food and Drug Administration (FDA) for eteplirsen for the treatment of DMD amenable to exon 51 skipping.” sarepta.com ↗ “FDA has filed the New Drug Application (NDA) for eteplirsen for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.” sarepta.com ↗ |
| Filed | US (FDA) | — | DMD-related muscular dystrophy | 2015-06-26 | “completion of the rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for eteplirsen” sarepta.com ↗ |
| Approved Indication not stated 2 milestones | |||||
| Refused (EMA) | EU (EMA) | Exondys | — | 2018-12-06 | europa.eu ↗ |
| Approved | US (FDA) | EXONDYS 51 | — | 2016-09-19 | fda.gov ↗ |
Trials 10
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/22 trials | ||||||
| Phase 1/2 | NCT00844597 | Jan 2009 → Jun 2010 | Duchenne muscular dystrophy | Sarepta Therapeutics, Inc. | Completed | No outcome recorded |
| Phase 1/2 | NCT00159250 | Oct 2007 → Dec 2008 | Duchenne muscular dystrophy | Imperial College London | Completed | No outcome recorded |
| Phase 26 trials · 2 met primary | ||||||
| Phase 2 | NCT03985878 | Jun 2019 → Aug 2022 | Duchenne muscular dystrophy | Sarepta Therapeutics, Inc. | Terminated | No outcome recorded |
| Phase 2 | NCT03218995 | Aug 2017 → Mar 2021 | Duchenne muscular dystrophy | Sarepta Therapeutics, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT02420379 | Jun 2015 → Dec 2018 | Duchenne muscular dystrophy | Sarepta Therapeutics, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT02286947 | Nov 2014 → Apr 2017 | Duchenne muscular dystrophy | Sarepta Therapeutics, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT01540409 | Feb 2012 → Apr 2016 | Duchenne muscular dystrophy | Sarepta Therapeutics, Inc. | Completed | Met primary |
| Phase 2 | NCT01396239 | Jul 2011 → Feb 2012 | Duchenne muscular dystrophy | Sarepta Therapeutics, Inc. | Completed | Met primary |
| Phase 32 trials | ||||||
| Phase 3 | NCT03992430 | Jul 2020 → Oct 2026 expected | Duchenne muscular dystrophy | Sarepta Therapeutics, Inc. | Active not recruiting | No outcome recorded |
| Phase 3 | NCT02255552 | Nov 2014 → Jun 2019 | Duchenne muscular dystrophy | Sarepta Therapeutics, Inc. | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 52
| Date | Issuer | Release |
|---|---|---|
| 2021-03-15 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics to Present Results from its Gene Therapy and RNA Platforms at the 2021 Annual MDA Clinical and Scientific Conference sarepta.com ↗
An analysis of time to loss of ambulation in patients taking eteplirsen, a phosphorodiamidate morpholino oligomer (PMO) for the treatment of Duchenne, compared to standard of care. |
| 2018-09-21 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Receives Negative CHMP Re-examination Opinion for Eteplirsen sarepta.com ↗
Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), has confirmed its 31 May 2018 negative opinion for a Conditional Marketing Application for eteplirsen. |
| 2018-06-01 | Sarepta Therapeutics, Inc. | Regulatory As Anticipated, Sarepta Receives Negative CHMP Opinion for EXONDYS® (eteplirsen) to Treat Patients with Duchenne Muscular Dystrophy in Europe sarepta.com ↗
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), has adopted a negative opinion for EXONDYS® (eteplirsen), as expected. |
| 2017-12-27 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Announces Publication of Long-Term Pulmonary Function of Eteplirsen-Treated Patients Compared to Natural History of Duchenne Muscular Dystrophy in The Journal of Neuromuscular Diseases sarepta.com ↗
A statistically significant and clinically meaningful reduction in pulmonary decline as measured by forced vital capacity percent predicted (FVC%p) was observed for eteplirsen-treated patients as compared to natural history data published in the scientific literature. |
| 2016-12-19 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces EMA Validation of Eteplirsen Authorization Application for Treatment of Duchenne Muscular Dystrophy Amenable to Exon Skipping 51 sarepta.com ↗
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage developer of innovative RNA-targeted therapeutics, today announced that the European Medicines Agency (EMA) validated the previously submitted Marketing Authorization application (MAA) for eteplirsen to treat Duchenne muscular dystrophy amenable to exon 51 skipping. |
| 2016-09-19 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces FDA Accelerated Approval of EXONDYS 51™ (eteplirsen) injection, an Exon Skipping Therapy to Treat Duchenne Muscular Dystrophy (DMD) Patients Amenable to Skipping Exon 51 sarepta.com ↗
today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for EXONDYS 51™ (eteplirsen) |
| 2016-06-06 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces FDA Request For Dystrophin Data Prior To Making A Decision on Eteplirsen NDA sarepta.com ↗
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has requested that Sarepta provide dystrophin data, as measured by western blot, from biopsies already obtained from the ongoing confirmatory study of eteplirsen (PROMOVI), as part of its ongoing evaluation of the eteplirsen New Drug Application (NDA). |
| 2016-05-25 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces FDA Will Not Complete the Review of the Eteplirsen New Drug Application By The PDUFA Date sarepta.com ↗
the U.S. Food and Drug Administration (FDA) has notified the Company that they are continuing their review and internal discussions related to our pending NDA for eteplirsen and will not be able to complete their work by the Prescription Drug User Fee Act (PDUFA) goal date of May 26, 2016. |
| 2016-04-25 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Issues Statement on Advisory Committee Outcome for Use of Eteplirsen in the Treatment of Duchenne Muscular Dystrophy sarepta.com ↗
The advisory committee voted 6-7 against the finding of substantial evidence from adequate and well controlled studies that show that eteplirsen induces production of dystrophin to a level that is reasonably likely to predict clinical benefit (FDA Question #2). |
| 2016-03-14 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces FDA Advisory Committee Meeting to Review Eteplirsen as a Treatment for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping sarepta.com ↗
today announced that the Peripheral and Central Nervous System (PCNS) Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) will review Sarepta’s New Drug Application (NDA) for eteplirsen on April 25, 2016. |
| 2016-02-08 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Receives Notification of PDUFA Extension for Eteplirsen sarepta.com ↗
In a notice received from the FDA, the Prescription Drug User Fee Act (PDUFA) date for eteplirsen has been extended to May 26, 2016. |
| 2016-01-20 | Sarepta Therapeutics, Inc. | Regulatory FDA Postpones Advisory Committee Meeting to Review Eteplirsen Due to Severe Weather Storm in the Washington D.C. Area sarepta.com ↗ |
| 2015-11-17 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Announces Publication of Positive Long-Term Safety and Efficacy Data for Eteplirsen in the Annals of Neurology sarepta.com ↗
The study found that at three years of treatment, patients experienced a slower rate of disease progression when compared to untreated matched historical controls and the investigational drug continued to be well-tolerated. |
| 2015-10-01 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Announces Additional Long-Term Efficacy and Safety Data from Pivotal Phase IIb Program of Eteplirsen for Treatment of Duchenne Muscular Dystrophy sarepta.com ↗
The data demonstrated that eteplirsen provided a statistically significant advantage of 151 meters in the ability of study participants to walk at three years, compared with external controls. |
| 2015-08-25 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces FDA Has Filed Eteplirsen NDA for the Potential Treatment of Duchenne Muscular Dystrophy for Patients Amenable to Exon 51 Skipping sarepta.com ↗
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has filed the New Drug Application (NDA) for eteplirsen for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. |
| 2015-08-21 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Receives Rare Pediatric Disease Designation From FDA for Eteplirsen for the Potential Treatment of Duchenne Muscular Dystrophy sarepta.com ↗
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for eteplirsen, a potential treatment for patients with Duchenne Muscular Dystrophy (DMD) who are amenable to skipping exon 51. |
| 2015-06-29 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Completes NDA Submission to FDA for Eteplirsen for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping sarepta.com ↗
today announced the completion of the rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for eteplirsen on June 26, 2015. |
| 2015-01-12 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Reports Long-Term Outcomes through 168 Weeks from Phase IIb Study of Eteplirsen in Duchenne Muscular Dystrophy sarepta.com ↗
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-based therapeutics, today announced data through Week 168 from Study 202, a Phase IIb open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). |
| 2014-10-27 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces Regulatory Update on Eteplirsen sarepta.com ↗
The FDA indicated that further discussion with Sarepta “will be necessary to determine what would constitute a complete NDA.” |
| 2014-07-10 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Reports Long-Term Outcomes Through 144 Weeks from Phase IIb Study of Eteplirsen in Duchenne Muscular Dystrophy sarepta.com ↗
today announced data through Week 144 from Study 202, a Phase IIb open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). |
| 2014-02-05 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Announces Eteplirsen Demonstrates Stability on Pulmonary Function Tests through 120 Weeks in Phase IIb Study in Duchenne Muscular Dystrophy sarepta.com ↗
Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced new pulmonary function data through Week 120 from Study 202, a Phase IIb open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). |
| 2014-01-15 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Announces Eteplirsen Demonstrates Continued Stability on Walking Test through 120 Weeks in Phase IIb Study in Duchenne Muscular Dystrophy sarepta.com ↗
Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced data through Week 120 from Study 202, a Phase IIb open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). |
| 2013-11-12 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces FDA Considers NDA Filing for Eteplirsen Premature in Light of Recent Competitive Drug Failure and Recent DMD Natural History Data sarepta.com ↗
As a result of these recent data, the FDA stated that they "currently consider an NDA filing for eteplirsen as premature." |
| 2013-09-26 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Announces Eteplirsen Demonstrates Continued Stability on Walking Test Through 96 Weeks in Phase IIb Study in Duchenne Muscular Dystrophy sarepta.com ↗
Results through nearly two years showed a continued stabilization of walking ability in eteplirsen-treated patients evaluable on the 6-minute walk test (6MWT). |
| 2013-08-08 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Announces Publication of Eteplirsen Clinical Study Results in the Annals of Neurology sarepta.com ↗
Published study results showed that once-weekly treatment with eteplirsen resulted in a statistically significant increase from baseline in novel dystrophin, the protein that is lacking in patients with DMD. |
| 2013-06-19 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Announces Eteplirsen Demonstrates a Continued Benefit on Walking Test Through 84 Weeks in Phase IIb Study in Duchenne Muscular Dystrophy sarepta.com ↗
Results at 84 weeks showed a continued stabilization of walking ability in eteplirsen-treated patients evaluable on the 6-minute walk test (6MWT). |
| 2013-04-05 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Announces Eteplirsen Demonstrates Sustained Benefit on Walking Test Through 74 Weeks in Phase IIb Study in Duchenne Muscular Dystrophy sarepta.com ↗
Results at 74 weeks showed a continued stabilization of walking ability in eteplirsen-treated patients evaluable on the 6-minute walk test (6MWT). |
| 2012-12-07 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Announces a Continued Benefit on Walking Test Through 62 Weeks in Phase IIb Open-Label Extension Study of Eteplirsen in Duchenne Muscular Dystrophy sarepta.com ↗
The eteplirsen treatment cohort (n=6) continued to show disease stabilization and the cohort has shown less than a 5% decline in walking distance on the 6-minute walk test from baseline. |
| 2012-10-12 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics to Present Additional 48-Week Data From the Phase IIb Study of Eteplirsen for the Treatment of Duchenne Muscular Dystrophy at the 17th Annual International World Muscle Society sarepta.com ↗
The presentation will describe new and previously reported efficacy and safety data from the Phase IIb study examining 48 weeks of treatment with eteplirsen in boys with DMD. |
| 2012-10-03 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Announces Eteplirsen Meets Primary Endpoint of Increased Novel Dystrophin and Achieves Significant Clinical Benefit on 6-Minute Walk Test After 48 Weeks of Treatment in Phase IIb Study in Duchenne Muscular Dystrophy sarepta.com ↗
Eteplirsen administered once weekly at either 30 mg/kg or 50 mg/kg for 48 weeks (n=8) resulted in a statistically significant increase (p≤0.001) in dystrophin-positive fibers to 47.0% of normal. |
| 2012-07-24 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Announces Significant Clinical Benefit With Eteplirsen After 36 Weeks in Phase IIb Study for the Treatment of Duchenne Muscular Dystrophy sarepta.com ↗
Sarepta Therapeutics (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced that treatment with its exon-skipping compound, eteplirsen, achieved a significant clinical benefit on the primary clinical outcome, the 6-minute walk test (6MWT), over a placebo/delayed treatment cohort in a Phase IIb trial in Duchenne muscular dystrophy (DMD) patients. |
| 2012-04-25 | Sarepta Therapeutics, Inc. | Results AVI BioPharma to Present Additional Data From the Phase IIb Study of Eteplirsen for the Treatment of Duchenne Muscular Dystrophy at the 2012 AAN Annual Meeting sarepta.com ↗
Results from the randomized, double-blind, placebo-controlled study confirm that the trial met its primary endpoint, demonstrating a significant increase in dystrophin at 24 weeks compared to placebo. |
| 2012-04-02 | Sarepta Therapeutics, Inc. | Results AVI BioPharma Announces Eteplirsen Meets Primary Endpoint, Demonstrating a Significant Increase in Dystrophin at 24 Weeks Compared to Placebo in Phase IIb Trial for the Treatment of Duchenne Muscular Dystrophy sarepta.com ↗ |
| 2012-01-04 | Sarepta Therapeutics, Inc. | Results AVI BioPharma Data Safety Monitoring Board Reports No Safety Concerns Identified in Phase IIb DMD Clinical Trial, Recommends Continuation of Trial sarepta.com ↗
Following its assessment of this biopsy data, along with other laboratory results and safety outcomes, the DSMB did not identify any safety concerns and determined it was safe to proceed with the trial as planned. |
| 2011-07-25 | Sarepta Therapeutics, Inc. | Results The Lancet Publishes Clinical Trial Data That Demonstrate Statistically Significant and Dose-Dependent Expression of Dystrophin in Duchenne Muscular Dystrophy Patients Treated With AVI BioPharma's Eteplirsen sarepta.com ↗ |
| 2011-04-11 | Sarepta Therapeutics, Inc. | Regulatory AVI BioPharma Receives Approval of Nonproprietary Name Eteplirsen for Lead Duchenne Muscular Dystrophy Therapeutic Candidate, AVI-4658 sarepta.com ↗ |
| 2010-10-15 | Sarepta Therapeutics, Inc. | Results AVI BioPharma's Investigational Drug Candidate AVI-4658 Demonstrates Broadly Favorable Profile of Safety and Tolerability, New Dystrophin Expression, Stable Clinical Performance and Inflammatory Modulation in the Treatment of Duchenne Muscular Dystrophy sarepta.com ↗ |
| 2010-07-07 | Sarepta Therapeutics, Inc. | Regulatory AVI BioPharma Opens Investigational New Drug (IND) Application for AVI-4658 in Duchenne Muscular Dystrophy sarepta.com ↗
AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based therapeutics, today announced that following review by the U.S. Food and Drug Administration the Company's Investigational New Drug (IND) application for AVI-4658 is open. |
| 2010-02-04 | Sarepta Therapeutics, Inc. | Results AVI BioPharma GLP Safety Pharmacology and Genotoxicity Evaluations of AVI-4658 for Duchenne Muscular Dystrophy Published in the International Journal of Toxicology sarepta.com ↗
It was also reported that the genotoxicity evaluation of AVI-4658 revealed no genotoxic potential, even at very high concentrations. |
| 2009-12-22 | Sarepta Therapeutics, Inc. | Results Systemic Treatment with AVI-4658 Demonstrates RNA Exon Skipping and Dystrophin Protein Expression in Duchenne Muscular Dystrophy Patients sarepta.com ↗
AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based drugs, today announced initial efficacy data from the ongoing Phase 1b/2 clinical trial of AVI-4658 for the systemic treatment of patients with Duchenne muscular dystrophy (DMD), a genetic muscle wasting disease caused by failure to produce dystrophin. |
| 2009-11-19 | Sarepta Therapeutics, Inc. | Results AVI BioPharma, Inc. Presents Update on AVI-4658 at TREAT-NMD / NIH International Conference sarepta.com ↗
The maximum cumulative dose administered to date is 3132 mg and the maximum single dose is 900 mg with no adverse safety signals, in either case. |
| 2009-10-26 | Sarepta Therapeutics, Inc. | Results AVI BioPharma Presents Updated Safety Data from Ongoing Systemic Trial of AVI-4658 at 7th Annual Action Duchenne International Conference sarepta.com ↗
There have been no safety issues identified, although one Serious Adverse Event was reported due to an anaesthetic-induced post-treatment biopsy procedure at 14 weeks, 2 weeks after last dose, causing nausea and vomiting. |
| 2009-10-20 | Sarepta Therapeutics, Inc. | Results Exon Skipping Drug Prevents Muscle Wasting and Maintains Muscle Function in Severely Affected, Dystrophin Deficient Mice sarepta.com ↗
An exon skipping PPMO has demonstrated dramatic effects in the prevention and treatment of severely affected, dystrophin and utrophin-deficient mice, preventing severe deterioration of the treated animals and extending their lifespan. |
| 2009-10-19 | Sarepta Therapeutics, Inc. | Results Exon Skipping Therapy Shows Promise in Prevention or Delay of Heart Disease Associated with Duchenne Muscular Dystrophy sarepta.com ↗
AVI BioPharma, Inc. (Nasdaq: AVII), a developer of RNA-based drugs, today announced the publication of research demonstrating the ability of a peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) therapy to prevent the onset of cardiomyopathy in a mouse model of Duchenne muscular dystrophy (DMD). |
| 2009-09-14 | Sarepta Therapeutics, Inc. | Results AVI BioPharma Presents Safety Data in Duchenne Muscular Dystrophy at 14th Annual International Congress of the World Muscle Society sarepta.com ↗
This safe and well-tolerated production of new dystrophin is believed to be the key to restoring muscle function and successfully treating patients with DMD - a condition for which there is no currently approved disease modifying therapy. |
| 2009-08-25 | Sarepta Therapeutics, Inc. | Results AVI BioPharma to Present Safety Data in Duchenne Muscular Dystrophy at 14th Annual International Congress of the World Muscle Society sarepta.com ↗
Data from the completed single-blind, placebo-controlled and dose escalation Phase 1 trial showed that AVI-4658 was safe when injected intramuscularly and successfully induced the production of dystrophin protein in patients in a dose-responsive manner. |
| 2009-08-25 | Sarepta Therapeutics, Inc. | Results AVI BioPharma Phase 1 Proof of Concept and Safety Data for AVI-4658 in Duchenne Muscular Dystrophy Featured in Lancet Neurology sarepta.com ↗
AVI BioPharma, Inc. (Nasdaq: AVII), a developer of RNA-based drugs, today announced that the results and scientific findings of its Phase 1 clinical trial assessing the "proof of concept" and safety of AVI-4658 in patients with Duchenne Muscular Dystrophy (DMD) have been published online in the journal, Lancet Neurology. |
| 2009-07-29 | Sarepta Therapeutics, Inc. | Results AVI BioPharma, Inc. to Present at IBC Drug Discovery & Development Conference: Oligonucleotide Therapeutics -- From Concept to Implementation sarepta.com ↗
Results from a single dose escalation clinical study of AVI-4658, a PMO therapeutic, demonstrate that a single intramuscular (IM) showed that injection of the drug into a small foot muscle of several DMD patients successfully induced dystrophin production in each patient |
| 2009-05-19 | Sarepta Therapeutics, Inc. | Results AVI BioPharma, Inc. to Present at TIDES 2009 Conference sarepta.com ↗
Results from a Phase 1 single dose escalation study of AVI–4658 testing the effect of intramuscular (IM) showed that injection of the drug into the muscles of a series of DMD patients successfully induced dystrophin production in each patient. |
| 2007-12-03 | Sarepta Therapeutics, Inc. | Regulatory AVI BioPharma Receives Fast Track Designation for AVI-4658 for Treatment of Duchenne Muscular Dystrophy sarepta.com ↗
AVI BioPharma, Inc. (Nasdaq:AVII), today announced that the U.S. Food and Drug Administration has granted Fast Track status to its product candidate, AVI-4658, for the treatment of Duchenne muscular dystrophy (DMD). |
| 2007-11-01 | Sarepta Therapeutics, Inc. | Regulatory AVI BioPharma Receives Orphan Drug Designation for AVI-4658 for Treatment of Duchenne Muscular Dystrophy sarepta.com ↗
AVI BioPharma, Inc. (Nasdaq:AVII), announced today that the U.S. Food and Drug Administration's (FDA) Office of Orphan Products Development has granted orphan drug designation to AVI-4658 for the treatment of Duchenne muscular dystrophy (DMD). |
| 2007-10-26 | Sarepta Therapeutics, Inc. | Regulatory AVI BioPharma Provides Update on Clinical Trial for Treatment of Duchenne Muscular Dystrophy sarepta.com ↗
Research teams at the Imperial College of London, in collaboration with the United Kingdom-based MDEX Consortium, have received approval from the Medicines and Healthcare products Regulatory Agency (MHRA) in the U.K. to begin screening patients for a proof-of-principle dose-escalating clinical trial using AVI-4658. |
All press releases naming this drug 70 releases
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 13 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Eteplirsen | “AVI-4658 (Eteplirsen)” NCT01396239 ↗ |
| Known as | AVI-4658 | “AVI-4658 (Eteplirsen)” NCT01540409 ↗ |
| Action | Restore | “the ability of AVI-4658 to induce exon 51 skipping and dystrophin restoration” PMID 21784508 ↗ Jul 2011 |
| Modality | Antisense oligonucleotide | “an intramuscular morpholino splice-switching oligonucleotide (AVI-4658) that skips exon 51 in dystrophin mRNA” PMID 19713152 ↗ Aug 2009 |
| Route | Intramuscular | “AVI-4658 was injected into the EDB muscle” PMID 19713152 ↗ Aug 2009 |
| Target | DMD | “designed to skip DMD exon 51” PMID 26573217 ↗ Jan 2016 |