FDA approves first gene therapy for hemophilia B

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

The U.S. Food and Drug Administration on Tuesday approved the first gene therapy to treat people with hemophilia B, an inherited bleeding disorder., writes STAT. The one-time treatment, called Hemgenix, was developed by Dutch biotech company UniQure and will be marketed by Australian pharmaceutical company CSL Behring. Hemgenix will cost $3.5 million, making it the most expensive drug approved to date. Hemgenix’s approval gives doctors 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 enough of a clotting protein called Factor IX.

Unlock this article by subscribing to STAT+ and enjoy your first 30 days for free!


Leave a Comment

Your email address will not be published. Required fields are marked *