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Sarepta Therapeutics Announces FDA Request For Dystrophin Data Prior To Making A Decision on Eteplirsen NDA
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun. 6, 2016-- 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). The Company plans to submit data from thirteen patient biopsy samples, at baseline and Week 48, to the FDA over the coming weeks to facilitate a prompt decision on the NDA by the Agency.
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Source: press release, 6/06/16. http://investorrelations.sarepta.com/phoenix.zhtml?c=64231&p=irol-newsAr...

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Sarepta Therapeutics Announces FDA Will Not Complete the Review of the Eteplirsen New Drug Application By The PDUFA Date
CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 25, 2016-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that 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. The FDA has communicated that they will continue to work past the PDUFA goal date and strive to complete their work in as timely a manner as possible.
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Source: press release, 5/25/16. http://investorrelations.sarepta.com/phoenix.zhtml?c=64231&p=irol-newsAr...

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Sarepta Therapeutics Receives Notification of PDUFA Extension for Eteplirsen

PDUFA goal date extended by standard extension period of three months to May 26, 2016

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Feb. 8, 2016-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the U.S. Food and Drug Administration (FDA) will require additional time to complete its review of the New Drug Application (NDA) for eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. In a notice received from the FDA, the Prescription Drug User Fee Act (PDUFA) date for eteplirsen has been extended to May 26, 2016. The rescheduled date for the Peripheral and Central Nervous System Advisory Committee meeting has not yet been determined.
The FDA notified Sarepta that its January 8, 2016 submission of 4-year clinical effectiveness data, which included additional six minute walk test (6MWT) and loss of ambulation data compared to a historical control, has been designated as a major amendment to the NDA. The FDA stated that the PDUFA goal date has been extended by three months to allow for a full review of the submission. As described in the Sarepta Advisory Committee Briefing Document Addendum, the principal basis for establishing the effectiveness of eteplirsen is a comparison of patients in Study 201/202 to a historical control group.
“While our primary goal is to bring treatment to patients with Duchenne as quickly as possible, we appreciate the efforts of the FDA to conduct a complete review of all of the data supporting our NDA and we remain committed to working closely with them throughout the remainder of the regulatory process”, said Edward Kaye, Sarepta’s interim chief executive officer and chief medical officer.
The FDA has previously granted eteplirsen Priority Review status, which is designated for drugs that 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 percent of people with the disease having mutations addressable by eteplirsen/exon 51 skipping.
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Source: press release, 2/08/16. http://investorrelations.sarepta.com/phoenix.zhtml?c=64231&p=irol-newsAr...

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Sarepta Therapeutics Announces FDA Has Filed Eteplirsen NDA for the Potential Treatment of Duchenne Muscular Dystrophy for Patients Amenable to Exon 51 Skipping
FDA Grants Priority Review Status

PDUFA Date is February 26, 2016

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Aug. 25, 2015-- 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. Approximately 13% of people with Duchenne muscular dystrophy are estimated to have a mutation addressable by Eteplirsen/exon 51 skipping.

The FDA has completed its filing review and has determined that our application is sufficiently complete to permit a substantive review. The Prescription Drug User Fee Act (PDUFA) action date for a decision on the application is February 26, 2015. The FDA has granted eteplirsen Priority Review status, which is designated to drugs that offer benefit over existing therapies, or provide a treatment where no adequate therapy exists.
“We are pleased with the FDA’s acceptance of our NDA for eteplirsen, as it represents an important milestone, not only for Sarepta, but for the Duchenne community. We look forward to continuing to work closely with the FDA during the regulatory review process,” said Edward M. Kaye, interim chief executive officer and chief medical officer. “We believe eteplirsen has the potential to make a meaningful impact on the lives of patients amenable to skipping exon 51 and we aim to build on our experience with eteplirsen to work with the FDA to inform and potentially expedite the clinical and regulatory pathway for the follow on exons, with the goal of reaching as many patients amenable to exon skipping as possible.”
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Source: press release, 8/25/15.

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Sarepta Therapeutics Completes NDA Submission to FDA for Eteplirsen for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun. 29, 2015-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, 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. Eteplirsen, the Company’s lead drug candidate, targets the underlying cause of Duchenne muscular dystrophy and is designed to enable the production of a functional internally truncated dystrophin protein in patients with mutations amenable to exon 51 skipping. Approximately 13% of people with Duchenne muscular dystrophy are estimated to have a mutation targeted by Eteplirsen/exon 51 skipping.

“The completion of our NDA submission for eteplirsen represents the culmination of the efforts of our employees, investigators, clinical trial sites, and most importantly the patients and families of the Duchenne community,” said Edward M. Kaye, interim chief executive officer and chief medical officer. “We look forward to working with the FDA during the regulatory process in pursuit of our goal of bringing eteplirsen to patients amenable to exon 51 skipping, while maintaining our organizational focus on advancing our PMO technology to target other DMD subpopulations amenable to exon-skipping as quickly as possible.”

The NDA submission includes a request for Priority Review. Previously, eteplirsen has been granted Orphan and Fast Track status by the FDA.
The rolling submission of the NDA began on May 20, 2015, after the completion of a pre-NDA meeting with the FDA held on May 19, 2015.
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Source: press release, 6/29/15. http://investorrelations.sarepta.com/phoenix.zhtml?c=64231&p=irol-newsAr...

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Sarepta Therapeutics Announces Plans to Submit Rolling NDA for Eteplirsen following Today’s Pre-NDA Meeting with the FDA

- Held pre-NDA meeting and agreed on rolling submission with the Agency -

- Will submit first two components of the NDA this week -

- Plans to complete the submission by mid-year -

CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 19, 2015-- Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the Company held a pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) regarding its lead product candidate, eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD). Sarepta has agreed with the Agency to initiate a rolling NDA submission and will submit the non-clinical and CMC components of the NDA by the end of this week. As previously announced, Sarepta plans to submit the final component of the NDA by mid-year 2015.

"We will initiate a rolling NDA submission to facilitate the regulatory review of the NDA,” said Edward Kaye, M.D., Sarepta’s interim chief executive officer and chief medical officer. “The initiation of our NDA submission for eteplirsen marks a significant milestone for the Duchenne community and we look forward to completing the submission by the middle of the year and to continuing to work with the Agency towards the goal of providing treatments to patients as quickly as possible.”
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Source: press release, 5/19/15. http://investorrelations.sarepta.com/phoenix.zhtml?c=64231&p=irol-newsAr...

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Ed Kaye, MD, SVP, Chief Medical Officer and Interim CEO, gave guidance for the completion of the rolling-NDA for Eteplirsen. He stated, "I would like to start by saying the team at Sarepta has remained highly focused and committed during the recent transition. As a result we remain on track towards our goal of compiling a complete NDA application for a plan submission in midyear 2015."
Source: Q1 2015 earnings conference call, 5/07/15.

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Sarepta Therapeutics Announces Regulatory Update on Eteplirsen

Updated and additional guidance received from FDA on specific data requirements for NDA;

FDA states further discussion needed to determine what constitutes a “complete” NDA submission;

NDA submission planned for mid-year 2015;

Company to hold teleconference today at 8:00 a.m. EDT

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Oct. 27, 2014-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-based therapeutics, today provided an update on its discussions with the U.S. Food and Drug Administration (FDA) regarding its planned New Drug Application (NDA) submission for the approval of eteplirsen for the treatment of Duchenne muscular dystrophy (DMD).

In meeting minutes received last week from a Type B Pre-NDA meeting that took place in September 2014, the FDA provided updated guidance regarding the specific data to be included as part of, or at the time of, Sarepta’s NDA submission. The guidance states that additional data are now required as part of the NDA submission, including the results from an independent assessment of dystrophin images and the 168-week clinical data from study 202. Additionally, the guidance requests more specific data including a minimum duration of safety in new patients exposed to eteplirsen, patient-level natural history data to be obtained by Sarepta from independent academic institutions, and MRI data from a recent study conducted by an independent academic group. The FDA indicated that further discussion with Sarepta “will be necessary to determine what would constitute a complete NDA.” Based on these requests, Sarepta plans to submit an NDA by mid-year 2015, pending any additional requests from further discussions with the FDA.

"We are committed to satisfying the FDA’s updated requests for these specific data to be included as part of an NDA submission and will continue to work with the Agency toward the goal of a complete and acceptable NDA filing," said Chris Garabedian, president and chief executive officer of Sarepta Therapeutics. "We believe all of the data requests and additional FDA discussions that have currently been outlined can be completed in time for an NDA submission by mid-year 2015. Obtaining an FDA approval of eteplirsen for the DMD patients who may benefit from the drug continues to be our highest priority.”

Excerpts from the Pre-NDA Meeting Minutes related to information that the FDA is requesting as part of an NDA submission included:

"The sponsor should include 3-month data from at least 12 to 24 newly exposed patients at the time the NDA is submitted."

"Available data from the other patients enrolled in the new eteplirsen studies (studies 301, 203, 204) should also be included at the time the NDA is submitted, even if exposure is less than 3 months in duration."

"Additional data from later time points and from newly enrolled patients should be submitted in the 120-Day Safety Update."

"FDA strongly advises the sponsor to obtain and submit patient-level natural history data. FDA is prepared to appeal to the academic groups holding the data to allow the sponsor a means to acquire the data."

"The study 201/202 clinical site inspection conducted in May, 2014, after the issuance of the April 15, 2014, guidance letter, uncovered marked disparities in the immunohistochemistry methodology and concerns about the reproducibility of the data. The lack of confirmation of robust dystrophin measurement during the site visit necessitates including the independent assessment of dystrophin-positive fibers and 168-week efficacy data from study 201/202 in the NDA."

“FDA strongly urged the sponsor to submit the MRI data with appropriate natural history controls.”

The FDA also stated that “[a]dditional discussion between the sponsor and the FDA will be necessary to determine what would constitute a complete NDA.”

Conference Call Information
Sarepta will hold a conference call to discuss this update today at 8:00 a.m. EDT (5:00 a.m. PDT). The conference call may be accessed by dialing 800.708.4539 for domestic callers and 847.619.6396 for international callers. The passcode for the call is 38376370. Please specify to the operator that you would like to join the "Sarepta Regulatory Update Call." The conference call will be webcast live under the investor relations section of Sarepta's website at www.sarepta.com and will be archived there following the call for 90 days. Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary. An audio replay will be available through November 3, 2014 by calling 888.843.7419 or 630.652.3042 and entering access code 38376370.
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Source: press release, 10/27/14. http://investorrelations.sarepta.com/phoenix.zhtml?c=64231&p=irol-newsAr...

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Sarepta Therapeutics Announces Plans to Submit New Drug Application to FDA for Eteplirsen for the Treatment of Duchenne Muscular Dystrophy by Year End 2014

-- FDA provides updated guidance on potential early approval pathway for eteplirsen;

-- Agreement reached with the Agency on eteplirsen open-label confirmatory studies with enrollment of a broader base of DMD patients later this year;

-- FDA provides initial guidance on development of follow-on DMD drug candidates;

-- Company to hold teleconference at 8:00 a.m. EDT

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Apr. 21, 2014-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-based therapeutics, today announced it plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) by the end of 2014 for the approval of eteplirsen for the treatment of Duchenne muscular dystrophy (DMD). Eteplirsen is Sarepta’s lead exon-skipping drug candidate in development for the treatment of patients with DMD who have a genotype amenable to skipping of exon 51.

The plan to submit an NDA for eteplirsen by the end of 2014 is based on a guidance letter from the Agency that proposed a strategy regarding the submission of an NDA for eteplirsen under a potential Accelerated Approval pathway and served as the final meeting minutes for four meetings that took place between November, 2013 and March, 2014. The Agency stated that “with additional data to support the efficacy and safety of eteplirsen for the treatment of DMD, an NDA should be fileable,” and outlined examples of additional data and analysis that, if positive, will be important to enhance the acceptability of an NDA filing by addressing areas of ongoing concern in the existing dataset. Additionally, the Agency provided clear guidance on an open-label, historically controlled confirmatory study of eteplirsen, as well as initial guidance on a placebo-controlled study of one or more follow-on DMD drug candidates, which, like the open-label study, could also be considered an acceptable confirmatory study to verify the clinical benefit of eteplirsen in the event of an accelerated approval.

“As we announce our plan to submit an eteplirsen NDA by the end of 2014, we are very pleased with the detailed guidance that the FDA has provided us on a potential eteplirsen approval pathway and their support of a historically controlled eteplirsen confirmatory study,” said Chris Garabedian, president and chief executive officer of Sarepta Therapeutics. “We also appreciate that the FDA shares our urgency in dosing a broader base of eteplirsen patients and has encouraged us to begin the clinical program with our follow-on exon-skipping drugs as soon as possible.”

Based on the Agency’s guidance, Sarepta plans to initiate several additional clinical studies with eteplirsen later this year in exon-51 amenable genotypes. These studies will include a clinical trial with predefined efficacy endpoints for ambulatory patients between the ages of 7 to 16 years who can walk a minimum distance, and two additional clinical trials that will evaluate safety and biomarkers in DMD patients younger than 7 years and DMD patients who have advanced in their disease progression to a point they cannot walk a minimum distance or have become non-ambulant. Additionally, Sarepta plans to initiate a placebo-controlled study with one or more of its follow-on DMD exon-skipping drug candidates by the end of the year.
“We are excited to have guidance from the FDA that allows us to move quickly into additional clinical trials with eteplirsen to confirm our current understanding of eteplirsen’s safety profile, its effect on dystrophin production, and its impact on clinical outcomes in DMD patients,” said Edward Kaye, M.D., senior vice president and chief medical officer of Sarepta Therapeutics. “We are particularly pleased that the FDA shares our interest in accelerating the clinical development of our follow-on exon-skipping drugs and we expect to initiate enrollment in this trial later this year.”
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Source: press release, 4/21/14. 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: IIb

Event Type: Regulatory FDA: PDUFA DATE

Dates: 2016-05-26

Results:

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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
-- EXONDYS 51™ the first DMD treatment approved in the US, targets dystrophin deficiency, the underlying cause of Duchenne --
--U.S. Commercial Launch planned to commence immediately--
--Conference call Scheduled for September 19, 2016, 4:00 p.m. EST--
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sep. 19, 2016-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for EXONDYS 51™ (eteplirsen) as a once weekly intravenous infusion of 30 milligrams per kilogram for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation in the DMD gene that is amenable to exon 51 skipping. This indication is based on an increase in dystrophin in skeletal muscles observed in some patients treated with EXONDYS 51. A clinical benefit of EXONDYS 51 has not been established. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. The most common adverse reactions compared to a placebo group were vomiting (38%) and balance disorder (38%) with contusion, excoriation, arthralgia, rash, catheter site pain, and upper respiratory tract infection also reported more frequently than placebo (≥ 10%).
“Today’s accelerated approval of EXONDYS 51 represents a major milestone in the treatment of Duchenne Muscular Dystrophy for patients amenable to skipping exon 51 by targeting the underlying genetic cause of the disease – the lack of the dystrophin protein,” said Edward Kaye, M.D., Sarepta’s interim chief executive officer and chief medical officer. “We are grateful to the many patients and investigators who participated in EXONDYS 51’s clinical studies. EXONDYS 51 represents the culmination of many years of work across our entire organization and the Duchenne community to address a critical unmet need by bringing this novel medicine to patients. We will continue to leverage what we have learned from EXONDYS 51 to facilitate future development of potential new treatments targeting additional exons with the goal of one day treating all DMD patients amenable to exon skipping.”
The underlying cause of Duchenne muscular dystrophy is a mutation or error in the gene for dystrophin, an essential protein involved in muscle fiber function. Certain genetic mutations in DMD involve the deletion of exons, which interrupt proper translation of the genetic code into protein.
Duchenne muscular dystrophy is a fatal genetic neuromuscular disorder affecting an estimated one in approximately every 3,500 – 5,000 males born worldwide. It is estimated that up to thirteen percent of people with DMD have mutations addressable by EXONDYS 51.
Patients and physicians can access information by visiting www.SareptAssist.com or calling 1-888-727-3782.
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Source: press release, 9/19/16. http://investorrelations.sarepta.com/phoenix.zhtml?c=64231&p=irol-newsAr...

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