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Sarepta Therapeutics Announces FDA Advisory Committee Meeting to Review Eteplirsen as a Treatment for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
--Advisory Committee Meeting Rescheduled for April 25, 2016--
--PDUFA date for eteplirsen is May 26, 2016--
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Mar. 14, 2016-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, 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. The Prescription Drug User Fee Act (PDUFA) action date for completion of FDA review of the eteplirsen is May 26, 2016.
The FDA has granted eteplirsen Priority Review status, which is designated for drugs which provide a treatment where no adequate therapy exists. The FDA also granted Rare Pediatric Disease Designation to eteplirsen, as well Orphan Drug Designation and Fast Track Status.
It is estimated that Duchenne muscular dystrophy affects approximately one in every 3,500 – 5,000 boys born worldwide, with 13% of people with the disease having mutations addressable by eteplirsen/exon 51 skipping.
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Source: press release, 3/14/16. http://investorrelations.sarepta.com/phoenix.zhtml?c=64231&p=irol-newsAr...

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Sarepta Therapeutics Has Not Yet Been Informed of a Rescheduled Date for the Peripheral and Central Nervous System Advisory Committee Meeting to Review Eteplirsen
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Source: press release, 2/25/16. http://investorrelations.sarepta.com/phoenix.zhtml?c=64231&p=irol-newsAr...

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FDA Postpones Advisory Committee Meeting to Review Eteplirsen Due to Severe Weather Storm in the Washington D.C. Area

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jan. 20, 2016-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the U.S. Food and Drug Administration’s (FDA) Peripheral and Central Nervous System Advisory Committee meeting scheduled for Friday, January 22 has been postponed by the FDA due to an anticipated severe winter snowstorm forecasted to hit the Washington D.C. area. A future meeting date will be announced in the Federal Register. In the event of a change in the February 26, 2016 PDUFA date, the company will provide an update at that time.
The Advisory Committee was scheduled to review Sarepta’s New Drug Application (NDA) for eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The new drug application (NDA) for eteplirsen is being reviewed under the FDA accelerated approval pathway, which allows early approval of a drug for a serious or life-threatening illness that offers a benefit over current treatments.
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Source: press release, 1/20/16. http://investorrelations.sarepta.com/phoenix.zhtml?c=64231&p=irol-newsAr...

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Sarepta Therapeutics Announces FDA Advisory Committee Meeting to Review Eteplirsen as a Treatment for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Advisory Committee Meeting Scheduled for January 22, 2016
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dec. 18, 2015-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) will review Sarepta’s New Drug Application (NDA) for eteplirsen, on January 22, 2016. The Prescription Drug User Fee Act (PDUFA) action date for completion of FDA review of the eteplirsen NDA is February 26, 2016.
It is estimated that DMD affects approximately one in every 3,500 – 5,000 boys born worldwide, with 13% of people with the disease having mutations addressable by eteplirsen/exon 51 skipping.
“Duchenne muscular dystrophy is a devastating disease with no currently approved treatment available to the patients and families it affects.” said Edward Kaye, M.D., Sarepta’s interim chief executive officer and chief medical officer. “We look forward to discussing the efficacy and safety data included in our NDA submission for eteplirsen with the Advisory Committee, with the ultimate goal of bringing a treatment to patients with Duchenne amenable to skipping exon 51.”
The FDA has granted eteplirsen Priority Review status, which is designated for drugs which provide a treatment where no adequate therapy exists. The FDA also granted Rare Pediatric Disease Designation to eteplirsen, as well Orphan Drug Designation and Fast Track Status.
More
Source: press release, 12/18/15. http://investorrelations.sarepta.com/phoenix.zhtml?c=64231&p=irol-newsAr...

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Sarepta Therapeutics Announces Tentative FDA Advisory Committee Meeting to Review Eteplirsen as a Treatment for Duchenne Muscular Dystrophy

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Oct. 14, 2015-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) is tentatively scheduled to review Sarepta’s New Drug Application (NDA) for eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, on January 22, 2016. There has been no formal communication to the company or Federal Register notice of a confirmed advisory committee meeting date.

It is estimated that DMD affects approximately one in every 3,500 boys born worldwide, with 13% of people with the disease having mutations addressable by eteplirsen/exon 51 skipping.

“We have been verbally informed by the FDA that we have a tentative date of January 22, 2016 for our advisory committee review of eteplirsen and our PDUFA date of February 26, 2016, remains unchanged.” said Edward Kaye, M.D., Sarepta’s interim chief executive officer and chief medical officer. “We look forward to discussing the efficacy and safety data included in our NDA submission for eteplirsen with the committee, with the ultimate goal of bringing treatment to more patients with Duchenne.”
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Source: press release, 10/14/15. http://investorrelations.sarepta.com/phoenix.zhtml?c=64231&p=irol-newsAr...

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Compound/DeviceSpecialtyIndicationCompound ClassTarget
Eteplirsen (AVI-4658)Medical GeneticsDuchenne Muscular DystrophyPhosphorodiamidate morpholino oligomers (PMO)Exon 51 of the Dystrophin gene

Mechanism of action: Eteplirsen (AVI-4658) is a phosphorodiamidate morpholino oligomers (PMO), a distinct class of oligonucleotide analogs, applied as a splice switching oligomer (SSO). The drug is intended to bind to exon 51 of the dystrophin gene thus causing it to be skipped. By skipping exon 51, Eteplirsen may allow the remaining exons of the Dystrophin gene to assemble thereby restoring the gene’s ability to make a shorter – but still functional – form of dystrophin. The phosphorodiamidate morpholino oligomers (PMOs) are a distinct class of oligonucleotide analogs. They bind to RNA and efficiently interfere with gene expression in a sequence-specific manner. Their non-ionic character combined with resistance to degradation in the body offer efficacy with minimal toxicity.

Phase of Development: Filed

Event Type: Regulatory FDA: Advisory Committee Meeting

Dates: 2016-04-25

Results:

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Sarepta Issues Statement on Advisory Committee Outcome for Use of Eteplirsen in the Treatment of Duchenne Muscular Dystrophy
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Apr. 25, 2016-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-based therapeutics, today announced that the U.S. Food and Drug Administration’s (FDA) Peripheral and Central Nervous System Advisory Committee (PCNSC) met to review the new drug application (NDA) for eteplirsen as a treatment for Duchenne muscular dystrophy amenable to exon 51 skipping. 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). The advisory committee voted 3 – 7, with three abstentions, against finding substantial evidence based on the clinical results of the single historically controlled study (Study 201/202) that eteplirsen is effective for treatment of DMD (FDA Question #7).
“We would like to thank the hundreds of patients and families who participated in the discussion today, underscoring the critical unmet need of people living with Duchenne.” said Edward Kaye, M.D., Sarepta’s interim chief executive officer and chief medical officer. “We appreciated the opportunity to present our data to the advisory committee panel and will continue to work with FDA as they complete their review of the eteplirsen NDA. Today more than ever, we remain committed to our mission of bringing a treatment to the Duchenne community.”
DMD is a rare degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. The FDA is not bound by the Advisory Committee's recommendation but takes its advice into consideration when reviewing New Drug and Biologic License Applications in general. The Prescription Drug User Fee Act (PDUFA) action date for completion of FDA review of eteplirsen is May 26, 2016.
In three additional voting questions, the panel voted 5 to 7, with one abstention, against whether decisions to administer the 6-minute walk test (vs. conclusions that the patient could no longer walk) were sufficiently objective and free of bias and subjective decision-making by patients, their caregivers, and/or health care professionals to allow for a valid comparison between patients in Study201/202 and an external control group (FDA Question #4). The panel voted on the impact of the North Star Ambulatory Assessment with one panel member voting that it strengthened the persuasiveness of the findings in Study 201/202, with five voting that it weakened the persuasiveness, and seven voting that it had no effect (FDA Question #5). The panel also voted on the impact of the other tests of physical performance (e.g., rise time, 10-meter run/walk) on the persuasiveness of the findings in Study 201/202, with the result of one panel member voting that they strengthened the persuasiveness, two voting that they weakened the persuasiveness, and ten voting that they had no effect (FDA Question # 6).
The PCNSC Advisory Committee recommendation was based on a review of results from the Phase IIb clinical program for eteplirsen (studies 201 and 202), long-term outcomes (through 168 weeks) from the Study 202 open-label extension study, as well as 4-year clinical effectiveness data based on a comparison of patients in Study 201/202 to a historical control group that was designated as a major amendment to the NDA in January 2016.
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Source: press release, 4/25/16. http://investorrelations.sarepta.com/phoenix.zhtml?c=64231&p=irol-newsAr...

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