And today 17 looks attractive. Losses, naturally, could be recovered, if one has enough cash and a zero doubts in Sarepta's future.
Timing is not what is usually been mentioned - "We hope that we can bring this work to the clinic in 18 months to 3 years. Certainly we don't expect it to take 10 years or even 5 years before we are treating patients."
'someone big standing behind FDA' - Isn't it reasonable to assume that it's a few pharmaceuticals?
Bravo, conrad. Our government is 'blind' to such crime. It's corrupted and its members are overpaid with donations from the interest groups. Among them are pharmaceuticals - poison-makers, that by all means keep medical science at bay.
By ED SILVERMAN
In an unusual move, the FDA last night released a statement to clarify mounting confusion over the status of a drug being developed to treat Duchenne muscular dystrophy, a rare and fatal disease that often afflicts young boys who typically do not live past 30 years of age.
The agency, in fact, was actually responding to a separate statement released earlier this week by Sarepta Therapeutics SRPT +2.80%, which hopes to market a drug called eteplirsen and had attempted to provide an update on the status of its marketing application with the agency.
But the company statement caused the stock to sink a hefty 32%. Why? Sarepta indicated there would be a six-month delay in seeking approval and disclosed language from its meetings with FDA staff that suggested irregularities with trial data. The phrase “marked disparities” was seen by investors as a red flag.
This amounted to yet another roller coaster ride for investors and families with ailing children. A year ago, the FDA told the drug maker its study data was insufficient for approval, and the stock fell 64% . In April, Sarepta indicated an application would be filed by the end of 2014, and the stock rose nearly 50%.
But the FDA statement, which the agency issued to mollify parents of DMD patients, suggests Sarepta executives may have misunderstood some of the key issues that agency staffers discussed with the drug maker, notably the timing for submitting trial data and concerns about fraud at a clinical trial site.
“Over the past several years, FDA has worked extensively with Sarepta on the development of eteplirsen, and provided guidance” about what “would be necessary to determine whether [the drug] is effective and supports the filing of a New Drug Application,” the agency wrote. The letter then reiterates some specifics that agency staffers communicated to Sarepta about submitting data.
As an example, the FDA notes that staffers “expressed willingness to conduct a ‘rolling review’ of Sarepta’s NDA. Under a rolling review, companies can submit, and FDA can review, portions of an application as they are completed. Once submission of all components is complete, the review clock begin.” The suggestion is that Sarepta may have misinterpreted the review timeline.
The FDA “provided Sarepta with detailed recommendations on how to improve analyses and FDA’s most recent advice was consistent with the advice provided after” a meeting last April, the FDA stated. Interestingly, the agency used the words ‘consistent’ and ‘consistently’ four times to describe its approach toward conveying its advice and guidance toward the Sarepta team.
A spokesman for Sarepta says the drug maker would not comment on the FDA letter.
The FDA missive is playing well on Wall Street, where Sarepta shares are rising again. But the chain of events, which R.W. Baird analyst Brian Skorney describes as “the strangest back and forth we’ve seen between a [drug maker] and regulators,” also raises questions about the extent to which the FDA has been precise enough in conveying its messages to both Sarepta and the DMD community.
Skorney, for instance, pointed to contrasting statements from FDA about concerns over trial data. FDA minutes from a meeting with Sarepta discussed “market disparities” and the need for an independent assessment, which he notes carries a “clear connotation that no one who has ever spent a week in a lab could misconstrue.”
But the agency statement last night only mentioned the need for additional analyses and no evidence of fraud. Skorney suggests the FDA should “bear its fair share of blame” for any confusion. We asked the FDA for comment and will update you accordingly.
Overall, the FDA statement issued last night “is far from a glowing endorsement of Sarepta and the agency’s frustration is palpable,” Skorney writes in his investor note. “However, given the concerns raised this week, we think the letter is actually a net positive as it reaffirms a path towards a New Drug Application filing.”
And “while the letter reads as a sharp criticism of the company’s management of information disclosure, it also provides comfort that the agency’s efforts to better understand the profile didn’t turn up anything to insinuate data manipulation. Following the FDA response, we’re beginning to think the whole delay isn’t a result of a guidance change but a misunderstanding of the… process and timeline.”
I'm surprised that there ts not a brilliant law firm that would take this case - dying children of the planet against corrupt FDA.
Rico, good point. The available information moves from the thick fog into the darkness, and from there into obscurity.
It is not a luck of knowledge. It is INTENT to diminish the value of the science, and low the price, - to commit an easy take over.
God point, Ruby. If take over was planed a long time ago - which is possible - what a minimum offer per share you imagine?
If car is mishandled - it crashed. If trane takes a wrong way - it's collapsed. If captain losses direction - his ship will get lost. If CEO loses clarity and leads the study into a vicious circle - he is fired.