STAT+: Pharmalittle: FDA approves first gene therapy for Duchenne muscular dystrophy; states join FTC to stop Amgen deal
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 is exceedingly modest. Rain or shine, we plan to promenade with the official mascots, catch up on our reading and make time for a listening party — the rotation will likely include this, this, this and this. And what about you? Now that summer has officially arrived, perhaps this is an opportunity to plan a well-deserved getaway. While you ponder the possibilities, you could get a head start by enjoying the great outdoors, hit a few good eateries, or maybe visit a museum or two. Or if energy levels are low, curl up in front of the telly. Well, whatever you do, have a grand time. But be safe. Enjoy, and see you soon…
The U.S. Food and Drug Administration granted conditional approval to the first gene therapy for Duchenne muscular dystrophy, although the agency restricted the treatment to younger patients, with additional data required to broaden its use, STAT reports. The therapy, called Elevydis, is made by Sarepta Therapeutics. The company will charge $3.2 million for the treatment, making it the second-most expensive drug in the U.S., behind a recently approved gene therapy for hemophilia. This was the first time the FDA has accepted a request from a drugmaker to grant a gene therapy accelerated approval, under which drugs are provisionally authorized based on a surrogate marker.
The U.S. Food and Drug Administration rejected an investigational treatment for NASH from Intercept Pharmaceuticals, derailing what would have been the first approved medicine for a prevalent liver disease, STAT tells us. The rejection made clear that reapplying for approval would require waiting about three years for a large, ongoing clinical trial to conclude. Instead, Intercept plans to cease its investments in NASH research, abandoning a field of study whose promise once made it a multibillion-dollar company. The rejection was widely expected after a May hearing in which independent FDA advisers voted the benefits of the drug did not outweigh its risks.
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