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STAT+: Pharmalittle: We’re reading about FDA views on a Duchenne drug, GSK cost savings, and more

Дата публикации: 28-07-2026 13:25:49

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

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By Ed Silverman

July 28, 2026

Pharmalot Columnist, Senior Writer

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.

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.

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