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STAT·10h ago·3 min read
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STAT+: Pharmalittle: We’re reading about FDA views on a Duchenne drug, GSK cost savings, and more

The FDA said a Capricor stem cell treatment for Duchenne muscular dystrophy did not meet a Phase 3 trial's objectives — contrary to company claims

Jul 28, 2026·read at STAT ↗RegulatoryNeutral

STAT PlusNewsletterPharmalot Pharmalittle: We’re reading about FDA views on a Duchenne drug, GSK cost savings, and more Biotechs are spending billions to cure a rare and little-known liver disorder Manage alerts for this article Email this article Share this article By Ed SilvermanJuly 28, 2026 Pharmalot Columnist, Senior Writer Ed Silverman[email protected]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 morning Pharmalittle newsletter and the afternoon Pharmalot newsletter. Top of the morning to you.

Gray skies are hovering over the Pharmalot campus right now, but our spirits remain sunny, nonetheless. Why? We will trot out a bit of insight from the Morning Mayor, who would say “Every new day should be unwrapped like a precious gift.” To celebrate the notion, we are brewing still more cups of stimulation and invite you to join us.

Our choice today is witch’s brew. Meanwhile, here are a few items of interest. Hope you have a smashing day and, of course, do stay in touch.

… The U.S. Food and Drug Administration said that a Capricor Therapeutics stem cell treatment for Duchenne muscular dystrophy did not meet the objectives of a Phase 3 trial — contrary to company claims last year, STAT says. Last December, Capricor said its drug, known as deramiocel, met both the primary and secondary endpoints in a large, randomized study.

It was a striking result in a fatal, childhood disease that has proven stubbornly difficult to treat, despite immense advances in genetic medicine. But in documents released Monday in advance of a hearing this week where FDA advisers will weigh in on the drug, the agency said the study fell short.Advertisement The world risks a resurgence of the HIV epidemic after international financing to combat the infectious disease suffered a “profound shock” last year, STAT writes, citing a new report from UNAIDS, the United Nations agency. Overall, government funding declined by more than $1.5 billion to $7.3 billion in 2025, an 18% drop and the lowest level in nearly two decades.

Prevention programs, in particular, have historically relied heavily on donor assistance in most regions, especially in sub-Saharan Africa, where it reached 83% two years ago. Although the U.S. was blamed for funding cuts, it still provides 74% of government donor funding.

STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus in-depth analysis, newsletters, premium events, and news alerts. Already have an account?

Log in Monthly $39 Totals $468 per year $39/month Get Started Totals $468 per year Starter $30 for 3 months, then $399/year $30 for 3 months Get Started Then $399/year Annual $399 Save 15% $399/year Get Started Save 15% 11+ Users Custom Savings start at 25%! Request A Quote Request A Quote Savings start at 25%! 2-10 Users $300 Annually per user $300/year Get Started $300 Annually per user View All Plans To read the rest of this story subscribe to STAT+.

Subscribe Log In pharmalittle, STAT+ Submit a correction requestReprints 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 morning Pharmalittle newsletter and the afternoon Pharmalot newsletter. Newsletter Understand how science, health policy, and medicine shape the world every day Recommended Pharmalot July 27, 2026 STAT Plus: HIV resurgence is feared as global funding to combat the disease falls 18% Pharmalot July 27, 2026 STAT Plus: Pharmalittle: We’re reading about a U.S.

peptide panel’s votes, Amgen scuffling with the FDA, and more Advertisement Pharmalot July 24, 2026 STAT Plus: Up and down the ladder: The latest comings and goings Pharmalot July 24, 2026 STAT Plus: Pharmalittle: We’re reading about an FDA panel and peptides, a Merck license for an HIV pill, and more Pharmalot July 24, 2026 STAT Plus: Merck strikes voluntary licensing deals in poor countries for an experimental HIV prevention pill Subscriber Picks

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