PPMD Community

Sarepta and their push for Accelerated Approval of Eteplirsen

I do not understand why this information is not here on this site, but people should be aware of Sarepta's push with the FDA to gain accelerated approval of their drug, eteplirsen, designed to skip exon 51.

Sarepta Therapeutics Announces FDA Will Consider Accelerated Approval for Eteplirsen After Further Review of Data on Dystrophin and Clinical Outcomes
Eteplirsen Manufacturing and Clinical Activities Continue as Planned

CAMBRIDGE, MA -- (Marketwired) -- 04/15/13 -- Sarepta Therapeutics, Inc. (NASDAQ: SRPT) today provided an update on its discussions with the U.S. Food and Drug Administration (FDA) regarding a potential application for accelerated approval of eteplirsen for the treatment of Duchenne muscular dystrophy (DMD). The FDA has requested that Sarepta provide additional information from the existing eteplirsen dataset to inform a decision on the acceptability of this dataset for a New Drug Application (NDA) filing under the Subpart H Accelerated Approval regulatory pathway. This feedback was provided in meeting minutes from Sarepta's End-of-Phase II meeting with the FDA's Division of Neurology Products that occurred last month.

Specifically, Sarepta received feedback on both the acceptability of dystrophin as a surrogate endpoint that would reasonably predict clinical benefit in DMD patients and the acceptability of the eteplirsen safety database for a Subpart H Accelerated Approval filing.

"We are encouraged by our interactions with the FDA and their Division of Neurology Products and we view their request for more data as a reflection of the thorough and comprehensive approach that the Agency takes in evaluating a new surrogate marker," said Chris Garabedian, president and chief executive officer of Sarepta Therapeutics. "We are confident that the method in which we've collected dystrophin, the degree and consistency of the dystrophin levels, and the supporting clinical data will provide the Agency the information it needs to determine if dystrophin is a feasible surrogate marker that is reasonably likely to predict clinical benefit."

At the End-of-Phase II Clinical Meeting in March, there was a productive discussion on the suitability of dystrophin as a surrogate marker, including a presentation by Sarepta detailing the methodologies used to analyze dystrophin in the studies and supportive data suggesting that the dystrophin produced is functional. As follow up to the discussion, and as reflected in the meeting minutes from the Division of Neurology Products, the Agency requested two written summaries: "a coherent and comprehensive summary to support dystrophin as a surrogate" and "a detailed discussion of all clinical outcomes in the eteplirsen study."

Furthermore, the meeting minutes contained the following statement:

"The Agency stated that they had not made a final decision regarding acceptability of the proposed Subpart H (Accelerated Approval) NDA filing, and that the Agency would consider the additional data submitted by the sponsor before making a final decision."

Sarepta also discussed the eteplirsen and phosphorodiamidate morpholino oligomer (PMO) safety database at the End-of-Phase II meeting and asked if the 38 patient eteplirsen safety database was sufficient for a Subpart H Accelerated Approval filing. While Sarepta will continue to collect long-term safety from the ongoing eteplirsen extension study for a potential submission, the meeting minutes stated that: "In the event (the Agency) agrees to file the Subpart H NDA submission, additional safety data to support approval could come from the first few months of the... pivotal confirmatory study."Sarepta still intends to begin dosing patients in a pivotal confirmatory study in the first quarter of 2014.

Sarepta is preparing to submit the dystrophin and clinical outcomes summaries and will be requesting a follow-up meeting with the FDA to discuss these later this quarter. As a result, the End-of-Phase II CMC meeting with the FDA is now expected to occur in the third quarter, however all eteplirsen manufacturing and clinical development activities continue as planned and are not anticipated to delay the potential timeline of an Accelerated Approval NDA submission.

Conference Call Information

Sarepta will hold a conference call to discuss this update today at 5:00 p.m. EDT. The conference call may be accessed by dialing 800.446.2782 for domestic callers and 847.413.3235 for international callers. The passcode for the call is 34696256. Please specify to the operator that you would like to join the "Sarepta Therapeutics Corporate Conference Call." The conference call will be webcast live under the events section of Sarepta's website at www.sareptatherapeutics.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 May 9, 2013 by calling 888.843.7419 or 630.652.3042 and entering access code 34696256.

Views: 480

Reply to This

Replies to This Discussion

Hi Noreen,

This info was posted here at PPMD via Facebook on Monday. Thanks for keeping the message going. Here is another article about how Sarepta wants to change the way we do business with the FDA, if you are interested.

http://www.raredr.com/front-page-legislation/articles/sarepta-wants...

 

Andrea

Can you direct me to where it was posted? I searched PPMD and cannot find it.  If I were new to the world of DMD and found this site, I would be in the dark regarding Sarepta's latest news.  I would think news of this type would be on your front page.

I agree with you.

http://www.facebook.com/27058624982/posts/155691631264479/ It was appeared here on May 15th in the news feed (latest activity),via PPMD FB, but not as a "permanent" post. Perhaps it got forgotten about because of what happened in Boston.  I re-found it by hitting the "more..." button  and scrolling through all the topics.

A.

 

For parents waiting on such information - the Boston bombing IS a back seat...!   

Eteplirsen Phase IIB study met the primary endpoint of increase in novel dystrophin

Eteplirsen Phase IIB study met the primary endpoint of increase in novel dystrophin produced defined as a % of dystrophin positive fibers
— Significant levels of dystrophin in all eteplirsen-treated patients after 24 weeks of treatment (average of 47.0% after 48 weeks of treatment and 38.3% after 24 weeks of treatment )
— Patients showed evidence of continued dystrophin production with a longer duration of treatment as demonstrated in the 30mg/kg cohort at weeks 24 and 48
• Eteplirsen maintained a statistically significant clinical benefit on the primary clinical outcome, the 6-minute walk test (6MWT), over a placebo/delayed treatment cohort through week 74 in the 6MWT-evaluable patient (mITT) population
— The eteplirsen treatment group continued to show disease stabilization with a less than 5% decline in walking distance on the 6-minute walk test from baseline
— The placebo/delayed-treatment cohort showed a change in the trajectory of the mean 6MWT decline at week 36, consistent with the likely timing of meaningful dystrophin production
— The placebo/delayed-treatment cohort demonstrated stability in walking distance from week 36 through week 74

SUMMARY (CONTINUED)
• Eteplirsen was well tolerated at both dose levels (up to 50mg/kg/wk over 74 weeks) with no clinical treatment-related adverse events.
— No treatment interruptions or discontinuations — No laboratory evidence of toxicity

Reply to Discussion

RSS

© 2018   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service