STAT+: Pharmalittle: FDA approvals will stop if U.S. defaults on its debts; DEA failed to punish wholesaler blamed in opioid crisis
The FDA delayed its decision on a gene therapy for Duchenne muscular dystrophy and is leaning toward clearing it for only a subset of younger kids.
Oh, what a beautiful morning. Yes, this is a cliché, but the saying is apt, given the crystal clear blue skies and deliciously cool breezes enveloping the Pharmalot campus. This is also perfect hunting weather for the official mascot and his soon-to-be permanent playmate, who are busy chasing squirrels and chipmunks. As for us, we are hunting for items of interest, a few of which we have gathered further down this screen for your enjoyment. This means, of course, that it is time to get cracking. So we are grabbing our latest cup of stimulation — our choice today is caramel apple — and digging in. Hope your own day goes well and you accomplish much. And as always, we invite you to keep in touch….
The U.S. Food and Drug Administration delayed its decision on a Sarepta Therapeutics gene therapy for Duchenne muscular dystrophy and is leaning toward clearing it for only a subset of younger kids with the disease, STAT writes. The FDA is now expected to rule on the application by June 22 instead of May 29. The agency may grant accelerated approval for 4-year-old and 5-year-old patients, although Sarepta asked the FDA to give the drug accelerated approval for all Duchenne patients who could still walk, which would have included most patients under the age of 10 and even some in their teens. An FDA advisory panel last month recommended approval for that broader range.
FDA Commissioner Robert Califf warned that approvals of new drugs and devices will come to a halt if the debt ceiling negotiations fail and the U.S. government defaults on its debt, Bloomberg News tells us. “We’ve got to figure out the debt ceiling,” said Califf. “If the government shuts down, I won’t be able to pay people to review the applications that are in play.” A default would mean the agency has no money to pay FDA scientific reviewers. Debt limit talks remain at an impasse over spending cuts, particularly those demanded by Republicans. U.S. Treasury Secretary Janet Yellen has said the government could run out of money by June 1.
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