Storyline
FDA Challenges Efficacy of Investigational Drug Candidates
The FDA has raised significant concerns about the efficacy of multiple investigational drug candidates ahead of regulatory hearings and meetings. The agency has questioned whether Capricor's deramiocel stem cell treatment for Duchenne muscular dystrophy and Replimune's RP1 skin cancer treatment met their primary trial endpoints or demonstrated sufficient clinical benefit for approval.
This is a long-running storyline that has developed over 9 days. The homepage highlights its most recent activity, so the outlet count there reflects the latest wave. The totals above cover the full run.
FDACapricor TherapeuticsReplimunederamiocelRP1
2026-08-05
- STAT+: Pharmalittle: We’re reading about a Medicare pilot and orphan drugs, Pfizer cutbacks, and more
- Opinion | Hold right industry accountable for unaffordable health care: Big Pharma
- CVS blows past estimates, hikes guidance as insurance unit continues to improve
- STAT+: Exemption of rare disease therapies in drug-pricing pilots would wipe out huge savings, analysis finds
2026-08-04
- 'Godzilla' weight-loss shot that's more powerful than Ozempic set to be offered to some patients ahead of FDA approval: Find out if you're eligible
- Eli Lilly is allowing more patients early access to its experimental weight loss drug
- STAT+: Startup promising to scale custom CRISPR therapies abruptly scraps plans
- STAT+: Three biotechs with three news updates
- STAT+: The latest orphan drug exemptions may not be warranted, analysis finds