FDA Says Capricor Duchenne Drug Fell Short of Goals
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- FDA said Capricor Therapeutics' stem cell treatment deramiocel for Duchenne muscular dystrophy did not meet Phase 3 trial objectives — contradicting the company's December claim that it hit both primary and secondary endpoints.
- The FDA documents were released Monday in advance of a hearing this week where agency advisers will weigh in on deramiocel, which targets a fatal childhood disease that has resisted treatment despite advances in genetic medicine.
- UNAIDS reported that government funding to combat HIV fell by more than $1.5 billion to $7.3 billion in 2025 — an 18% drop and the lowest level in nearly two decades.
- The U.S. was blamed for the funding cuts yet still provides 74% of government donor funding, according to the UNAIDS report cited by STAT.
- Donor assistance accounted for 83% of HIV prevention program funding in sub-Saharan Africa two years ago, per UNAIDS data.
Why it matters: The FDA's public contradiction of Capricor's headline trial result — a drug for a fatal childhood disease with few treatment options — raises the stakes for this week's advisory panel vote and could reshape deramiocel's regulatory path. Separately, the 18% plunge in global HIV funding to a two-decade low signals concrete erosion of prevention programs that depended on donors, particularly in sub-Saharan Africa where 83% of prevention funding came from external sources.
