STAT+: Pharmalittle: We’re reading about FDA approval for Sarepta drug, MSF closing access campaign and more
And so, another working week will soon draw to a close. Not a moment too soon, yes? This is, you may recall, our treasured signal to daydream about weekend plans. Our agenda, so far, appears quite modest. We hope to tidy up around the castle, promenade with the official mascots and hold another listening party, where the rotation will likely include this, this, this, this and this. And what about you? Given the ongoing heat on this side of the pond, this may be a good time to visit a beach or lake. Conversely, you could avoid people and remain indoors to binge watch something on the telly, assuming the air conditioning works. Or you could plan a summer getaway. Well, whatever you do, have a grand time. But be safe. Enjoy, and see you soon…
In a surprise move, Doctors Without Borders is closing down its access-to-medicines campaign, which has been credited with ensuring needed drugs and vaccines have been made available to countless patients in low-income countries around the world, STAT reports. The organization plans to close its campaign by the end of this year and create a new effort devoted to access to products for health care. But unlike its access-to-medicines campaign, the new structure will primarily focus on products — medicines and vaccines — that Doctors Without Borders expects to need for its own relief efforts. A strategic plan for the new structure is not finalized, though, according to sources.
For a third time, Sarepta Therapeutics has convinced a top U.S. Food and Drug Administration official to overrule the prevailing view of their staff and approve a drug for Duchenne muscular dystrophy, STAT tells us. On Thursday evening, the agency announced it expanded the approval of Elevidys, Sarepta’s Duchenne gene therapy, to cover nearly all patients, regardless of age or wheelchair status, despite the fact that the drug failed a large, Phase 3 trial last year. That decision was made almost exclusively by Peter Marks, the director of the Center for Biologics Evaluation and Research, who overruled three review teams and two top lieutenants.
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