Pharmalittle: We're reading about another AstraZeneca trial failure, a Novo name change, and more

ByEd Silverman

Sept. 14, 2026

Pharmalot Columnist, Senior Writer

Ed Silverman

Ed’s stories explore prescription drug pricing, affordability and access, as well issues surrounding patents, litigation, and legislation. He is also the author of the morningPharmalittle newsletterand the afternoonPharmalot newsletter.

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,STATnotes. 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 approvalin 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.

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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,STATwrites. 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.

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1. Ed Silverman

   Pharmalot Columnist, Senior Writer

   Ed’s stories explore prescription drug pricing, affordability and access, as well issues surrounding patents, litigation, and legislation. He is also the author of the morningPharmalittle newsletterand the afternoonPharmalot newsletter.

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