Sarepta Obtains Faster Track at FDA
In a favorable move for Sarepta Therapeutics, the Food and Drug Administration reversed itself and agreed to consider the company's Duchenne muscular dystrophy drug for accelerated approval.
Monday's news prompted a 50% gain in Sarepta shares (ticker: SRPT) to $36.70 on the week. There still could be a lot of upside potential with Sarepta. If approved by the FDA, the drug, eteplirsen, could generate $500 million in annual U.S. sales and ultimately form the basis for a family of DMD drugs with a few billion dollars of annual domestic sales. Sarepta's market value is $1.5 billion. Companies like Alexion Pharmaceuticals (ALXN) that have drugs for rare diseases can be valued at 10 times sales, or more. Barron's has been bullish on Sarepta, including an article last week ("Sarepta: High-Profile Decision for the FDA") in which we argued that Wall Street was too pessimistic on the prospects for speedier approval.
Sarepta said it plans to file under the accelerated process by year end, which could mean FDA approval by mid-2015. DMD is an inherited, fatal, muscle-wasting disease affecting boys that usually puts them in wheelchairs by their early teens and results in death by age 20 or 25. It's caused by a lack of dystrophin, a protein critical to muscle health. Eteplirsen is designed to force cellular machinery to produce a truncated but functional form of the protein.
The FDA outlined two potential pathways to accelerated approval: BUT BOTH OF THIS OPTION IS OUT OF QUESTION EVEN FOR NDA FILLING AS PAST GSK EXPERIENCE -
“1. The clinical data from Study 201/202 [Phase IIb clinical trial program] on 6-minute walk could be considered a finding on an intermediate clinical endpoint that could have the potential to support accelerated approval.”
Related to this first pathway to Accelerated Approval, the Agency stated that they have “significant concerns regarding our ability to draw valid conclusions based on the Study 201/202 data with respect to walking performance and other data,” and identified areas relating to the interpretation of the existing data set that will be addressed as part of an NDA review once the NDA is filed.
“2. We have discussed the possibility of using a number of modalities to quantify dystrophin in muscle biopsies, and discussed how these biomarkers might be used as a surrogate endpoint(s) to support accelerated approval.”
In evaluating this pathway, the FDA expressed concerns about methodological problems in the assessments of dystrophin and, “remain skeptical about the persuasiveness of the (dystrophin) data” and, as a result, the Agency is “uncertain whether the existing dystrophin biomarker data will be persuasive enough to serve as a surrogate endpoint that is reasonably likely to predict clinical benefit." However, the Agency further states that if they “were to find the biomarker data to be adequate upon detailed review, however, they would have the potential to support accelerated approval.” To that end, the Agency proposed “a collaborative effort in which we will work to better un
OBTAINED ? No FDA showed them the correct way. They are bluffing with P2 findings of 2 patients from total 12 and that all they have for arguments with FDA, Even if SRPT planed to conduct more trials its not simple as CEO thinks. They have not done this for years and knows were well that it doesn't work, it will never. Other two pharma companies are already at verge of results from their phase-3 and then chapter is closed for SRPT for ever.
Here's part II
Baird analyst Brian Skorney, who lifted his price target to $53 from $35, wrote that the situation is "commercially ideal" because "the patient population is readily identified, the unmet medical need is high, the treatment is likely to be lifelong, and the market will be insensitive to price." Eteplirsen could cost up to $500,000 a year per patient.
Skepticism remains about eteplirsen and whether it will get approved. Yet, data from its clinical trial and ongoing anecdotal reports suggest that the drug is stabilizing the disease. Sarepta is risky because its financial outlook hinges on eteplirsen. But if the drug gets approved next year, Sarepta could trade at $100.
Sorry for thumbs down. Should have been a thumbs up! These boxes are too small on this damn I-pad. Yes this is great news and as Thig pointed out it's double whammy weekend for the shorts. Have to say I have a big grin on my face this am.
Also lot's going on in the UK. They could use our help!!!