Wired News – CHMP Adopted Negative Opinion for Sarepta’s EXONDYS in Europe to Treat Patients with Duchenne Muscular Dystrophy

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LONDON, UK / ACCESSWIRE / June 5, 2018 / If you want access to our free research report on Sarepta Therapeutics, Inc. (NASDAQ: SRPT), all you need to do is sign up now by clicking the following link www.active-investors.com/registration-sg/?symbol=SRPT as the Company’s latest news hit the wire. On June 01, 2018, the Company announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion against approval for EXONDYS® (eteplirsen) for treatment of patients with Duchenne muscular dystrophy (DMD). Register today and get access to over 1,000 Free Research Reports by joining our site below:

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Sarepta to Seek Re-Examination of the Opinion

Sarepta will request a re-examination of the opinion, which will result in the assignment of a new rapporteur and co-rapporteur. The Company will also request a Scientific Advisory Group (SAG) on DMD be called so that neuromuscular specialists, experienced with working with treatments for these patients, can provide expert guidance and insight into, among other things, the validity of the external controls used and the importance of certain functional endpoints, including for instance, the relevance of meaningful slowing pulmonary decline in patients with this difficult to treat disease. The re-examination process is expected to be completed by end of 2018.

Sarepta Expected the Negative Opinion

As reported on May 05, 2018 earnings call, the Company anticipated a negative opinion following the oral explanation trend vote. Based on discussions with CHMP representatives, it was clear that the CHMP did not conclude that eteplirsen is ineffective for exon 51 amenable patients, but rather that Sarepta did not meet the regulatory threshold for conditional approval, in part due to the use of external controls as comparators in the studies.

FDA Granted Accelerated Approval to EXONDYS® to Treat DMD Patients

In September 2016, the US Food and Drug Administration (FDA) granted accelerated approval for EXONDYS® as a once weekly intravenous infusion of 30 milligrams per kilogram for the treatment of DMD in patients who have a confirmed mutation in the DMD gene that is amenable to exon 51 skipping. This indication was based on an increase in dystrophin in skeletal muscles observed in some patients treated with EXONDYS®. Continued approval for this indication was contingent upon verification of a clinical benefit in confirmatory trials.

About Duchenne Muscular Dystrophy

DMD is a genetic disorder characterized by progressive muscle degeneration and weakness. It is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. Symptom onset is in early childhood, usually between ages 3 and 5, and leads to death, generally before the age of 30, most commonly from heart failure. The disease primarily affects boys, but in rare cases it can affect girls.

About EXONDYS®

EXONDYS® (eteplirsen) is being developed to treat individuals who have a specific type of DMD, comprising 13% of the overall population who have one of the mutations amenable to exon 51 skipping. If approved, it would be the first injectable therapy in Europe designed to treat the underlying cause of DMD, the lack of a protein called dystrophin. EXONDYS® utilizes Sarepta’s precision RNA splicing technology to correct specific genetic mutations and produce a shortened, but functional, form of the dystrophin protein.

About Sarepta Therapeutics, Inc.

Headquartered in Cambridge, Massachusetts, Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying DMD drug candidates.

Stock Performance Snapshot

June 04, 2018 – At Monday’s closing bell, Sarepta Therapeutics’ stock rose 2.22%, ending the trading session at $97.98.

Volume traded for the day: 1.29 million shares.

Stock performance in the last month – up 25.34%; previous three-month period – up 37.13%; past twelve-month period – up 212.34%; and year-to-date – up 76.10%

After yesterday’s close, Sarepta Therapeutics’ market cap was at $6.29 billion.

The stock is part of the Healthcare sector, categorized under the Biotechnology industry. This sector was up 0.3% at the end of the session.

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