Pharmalittle: AstraZeneca cancer pill fails, Novo changes name

Good morning, everyone, and welcome to another working week. We hope the weekend respite was relaxing and invigorating. Now, though, that oh-too-familiar routine of meetings, deadlines, and the like has returned with a vengeance. You knew this would happen, yes? To cope, we are relying, as always, on a cuppa stimulation. Our choice today is blueberry hibiscus. Feel free to join us. Remember, no prescription or prior authorization is required. Meanwhile, here are a few items of interest. Best of luck accomplishing your goals, and we hope you conquer the world. And, of course, do keep in touch. …

AstraZeneca disclosed that a breast cancer pill failed in a pivotal trial testing it as a first-line therapy in advanced tumors, a result that could limit the use of the medicine, STAT notes. The drug, camizestrant, in combination with another medicine did not outperform standard treatment on a measure of how long patients went before their cancer progressed. The drug earlier this month won accelerated approval in the U.S. as a treatment for patients who develop a mutation in their tumors that’s a sign of emerging drug resistance. Had the trial been successful, it could have opened up a much larger market for the drug. 

Scholar Rock received U.S. Food and Drug Administration approval for the first-ever therapy that targets the loss of muscle in spinal muscle atrophy, raising hopes that patients with the rare neurological disorder might have a better chance of moving and walking independently, STAT writes. The FDA approved the drug, Isembyld, for use in adults and children 2 years and older who are currently receiving SMA therapies that target SMN2, a key gene for neurons that control movement. The approval marks another step forward for patients with spinal muscular atrophy, a rare and progressive disease that destroys nerve cells that control speaking, breathing, and walking. Babies who go untreated usually die by the age of 2.