Pharmalittle: We’re reading about a CRISPR approval, selling meds directly to patients, and more

By Ed Silverman Jan. 17, 2024

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Alex Hogan/STAT

Rise and shine, everyone. The middle of the week is upon us. Have heart, though. You made it this far, so why not hang on for another couple of days, yes? And what better way to make the time fly than to keep busy. So grab that cup of stimulation — our flavor today is butter pecan, a household favorite — and get started. Meanwhile, do keep us in mind if you hear anything interesting. We hope you have a simply smashing day and emerge unscathed. …

The U.S. Food and Drug Administration approved a CRISPR-based medicine to treat beta thalassemia, an inherited blood disorder, STAT notes. This is the same potentially curative therapy that was cleared by the agency last month to treat sickle cell disease. The expanded approval of the therapy, called Casgevy, was widely expected but came ahead of a March 30 FDA deadline for a decision. The therapy, which was developed by Vertex Pharmaceuticals and CRISPR Therapeutics, requires administration through authorized treatment centers with experience in stem cell transplantation, and will soon be made available at a $2.2 million list price in the U.S, for both approved indications.

Eli Lilly plans to launch its weight-loss drug Mounjaro in new countries this year as it expands manufacturing capacity, Reuters writes. Mounjaro was approved in the U.S., U.K., and Europe late last year for weight loss, but has only been available — as Zepbound — in the U.S. In Europe, each country has the final say on whether to approve it for its population. Lilly is waiting for European approval for its pen injector, which delivers the medicine, and is “optimistic” that could happen by the middle of the year. Europe has approved the drug to be sold in vials. With soaring demand, supplies have been tight and Lilly has made deals to expand manufacturing capacity.

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