STAT+: Pharmalittle: FDA approves first gene therapy for hemophilia B; pharma’s reputation slips in U.S.

Happy Thanksgiving Eve from STAT reporter Andrew Joseph, filling in for the day. A note that this newsletter is taking a break Thursday and Friday, something to be thankful for indeed. Now, how to leave you before the big day? Sir Pharmalot himself suggested we make a mention of where turkey futures stand this morning, in case anyone is trying to score a last-minute turkey at bargain prices. Instead, we’re more focused on gaming out our oven strategy for the big meal, with a particular priority on the pecan pie we’re on tap for. We are, after all, a former winner of STAT’s pie contest, so we have a reputation to uphold. A few more pieces of wisdom as we head into the holiday: Warm up appropriately for both your turkey trots and turkey feasts — we don’t want any injuries out there. Salads absolutely have a place on the Thanksgiving table (you need something bright and acidic to cut through everything else that’s rich and fatty; cranberry sauce should not have to shoulder that responsibility alone). And finally, no matter how full you get, there’s always room for dessert — and yes, for an extra helping of gratitude. Enjoy the holiday.

The U.S. Food and Drug Administration on Tuesday approved the first gene therapy to treat people with hemophilia B, an inherited bleeding disorder, STAT writes. The one-time treatment, called Hemgenix, was developed by the Dutch biotech company UniQure and will be marketed by CSL Behring, an Australian pharmaceutical company. Hemgenix will cost $3.5 million, making it the most expensive drug approved to date. The approval of Hemgenix provides physicians with a new, potentially curative treatment option for patients with severe hemophilia B, a rare bleeding disorder caused by a genetic mutation that prevents the body from producing sufficient quantities of a clotting protein called Factor IX.

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