Everything that happens at Sarepta – real or imagined – is grounds for debate and rampant speculation on Twitter. So it’s no surprise that when Baird analyst Brian Skorney issued a note late last week saying that the FDA’s decision on Sarepta’s controversial drug eteplirsen is “imminent,” the online kerfuffle that followed included plenty of taunts, tantrums and tears.
Some necessary background: Sarepta’s NDA for eteplirsen has famously been delayed twice. The first delay came after FDA insiders roundly criticized the drug for failing to show signs of efficacy. The second came with their second salvo that took apart Sarepta’s counterattack on a point-by-point basis. Both reviews castigated the biotech for failing to heed repeated advice on running a bigger, better study. And then there was an expert panel vote against an approval, based on the agency’s overwhelmingly negative view.
For any other drug at any other company, that would have been it. The CRO letter would have been a foregone conclusion.
But that’s not Sarepta ($SRPT).
The company – which carefully integrated the full-throated support of the patient community with some of the most influential advocates in DC — has defied repeated forecasts of a likely rejection only to be given another chance. Their third chance has come in the form of an FDA request for biopsy information on 13 patients. And the general belief is that if the company can offer biomarker evidence that the drug is producing the dystrophin these boys lack, the agency will go ahead with an accelerated approval.
“We believe that right now, someone, whether at Sarepta or the FDA, is looking at baseline and 48-week Western blots for 13 PROMOVI patients,” Skorney noted. “At this point, we think the decision should be pretty cut and dry: if the baseline blots show no band, and the vast majority of the 48-week blots show a (dystrophin) band, we predict a rapid accelerated approval for eteplirsen.”
Essentially, the FDA has been creating a new accelerated approval pathway for drugs that stir widespread support tied to persistent lobbying efforts. Whether they give Sarepta a green light now or not, few biotechs – especially in rare, lethal diseases – will ignore the role of a passionate and motivated patient population in garnering special regulatory consideration for their treatment.
Whether that’s a good or a bad thing will be the focus on plenty more outraged Tweets in the days, months and years ahead.
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John Carroll, Editor and Co-Founder
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