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Sarepta Therapeutics, Inc. Message Board

  • riskon4life riskon4life Oct 19, 2012 2:10 AM Flag

    Accelerated Approval is Imminent

    Source: Cure Duchenne PR, Oct 3, 2012

    Duchenne Muscular Dystrophy is Closer to a Treatment with New Results from Sarepta Therapeutics, Cure Duchenne Early Supporter of Study

    Newport Beach, Calif.“We have personally seen the positive impact this drug has had on our son,” said Jenn McNary who has two sons with Duchenne. Ten-year-old Max is one of the boys enrolled in the Sarepta trial but his 13-year-old brother Austin was not eligible for the trial. “Max has shown significant improvement and is stronger while Austin is getting weaker. We’re hoping these results will allow Sarepta to get approval quicker so other Duchenne boys can benefit from this treatment that helps slow the progression of the disease.”

    Accelerated approval is indispensable for DMD patients. All parties, including Serepta, The FDA, and major advocacy/funding groups - such as Cure Duchenne - acknowledge the urgency. In my opinion, the FDA will do anything in their power to assure that this drug is expedited, through the formulation of a special protocol that will ensure the fastest path to approval for eteplirsen; likely, faster than any other drug in recent history.

    These unprecedented actions will be seen in an extremely positive light; a welcomed change for the FDA. Moreover, these cognizant actions will help alleviate relentless pressure from recent governmental bodies and numerous public advocacy groups that have unremittingly accused the FDA of moving "too slowly" and not giving deprived patients the life-saving drugs they so desperately need to prolong deadly illnesses or, ultimately, save lives.

    The key, for Sarepta , amongst other things, is to remain focused on procuring additional manufacturing and expansion capabilities, which will allow them to yield greater quantities of eteplirsen in the shortest period of time; this, whilst maintaining a strict QC protocol for the drug. This is paramount, in my opinion, because without the drug, kids have no access to treatment. Unfortunately, quantities of the drug are relatively limited at the moment (Manufactured on a small scale and primarily for trials only).

    The company has mentioned that they are working on details to expand production beyond the current "essential" quantities that are required for ongoing and future studies. By their own admission, Sarepta has been inundated by patients' families inquiring about individual access to the drug. This is another clue as to why the FDA is under enormous pressure to approve eteplirsen quickly. Many affected families and the nation’s most influential DMD Non-Profit institutions are already meeting with FDA officials and pleading their cases for accelerated approval and quick access to eteplirsen for qualified patients.

    To conclude, everything should materialize in the end, so I encourage investors to be patient. Sarepta is about to change the way we treat DMD and eteplirsen will go down as one of the biggest biotechnological breakthroughs in recent history. Stay the course, and always remain optimistic -- primarily for the sake of the kids in need.

    Good luck to all!

    Disclosure: Long SRPT. Not investment advice.

    Sentiment: Strong Buy

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