STAT+: Pharmalittle: U.K. approves world’s first CRISPR-based medicine; FDA to review pricey cancer drug lacking confirmatory data
U.K. regulators approved a CRISPR-based medicine to treat sickle cell disease and beta thalassemia, ushering in a new era of genetic therapy.
Rise and shine, everyone, another busy day is on the way. We can tell because the official mascots are racing about the Pharmalot grounds and the parade of vehicles outside our window is picking up. As for us, we are dutifully firing up the coffee kettle to brew another cup of needed stimulation. Our choice today is butter pecan. Please feel free to join us. Now, though, the time has come to get cracking. So here is the latest assembly line of items of interest for your enjoyment. We hope you find these useful and have a smashing day. Best of luck and, as always, do stay in touch. …
U.K. regulators approved a CRISPR-based medicine to treat both sickle cell disease and beta thalassemia, making it the world’s first therapy built on the revolutionary gene-editing technology and ushering in a new era of genetic medicine, STAT reports. The authorization of the therapy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is itself not a surprise. Studies have shown the one-time treatment enabled many sickle cell patients to live free of debilitating pain crises and relieved thalassemia patients of needing regular blood transfusions, raising hopes they have been effectively cured. But it also stands as a major achievement, coming just over a decade after the first lab experiments that showed CRISPR’s potential as a gene-editing tool were published.
Panels in both the U.S. House and U.S. Senate passed restrictions on pharmacy benefit manager practices, increasing the chances the measures will be included in future government spending bills, STAT says. The policies are not identical, but at least three PBM reforms have been passed by panels in both chambers. One would replace PBM fees tied to a percentage of drug prices with flat fees. Another would require Medicare to create standard performance measures on which PBMs base pharmacy payments. A third would require PBMs to report data about drug costs, savings, beneficiary out-of-pocket costs, payments to pharmacies, and the amount of business directed to PBM-owned pharmacies.
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