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9 Out of 12 Experts Said No to the Only Cell Therapy for Duchenne Heart Disease
An FDA advisory panel voted 9 to 3 that Capricor Therapeutics' deramiocel lacks substantial evidence of effectiveness for treating cardiomyopathy in Duchenne muscular dystrophy. The core problem: Capricor changed its statistical analysis plan after the study was done, and the FDA says the original plan didn't show a significant difference from placebo. Reviewers also flagged that patients' hearts were essentially normal when they entered the trial, raising the question of whether cardiomyopathy was even being treated. The formal decision comes August 22, but history suggests the agency rarely overrides a vote this lopsided.
Why it matters: Duchenne cardiomyopathy is the leading killer of DMD patients, and no approved therapy specifically targets it. This vote likely delays the first dedicated treatment for years, leaving families with only borrowed heart-failure drugs while the FDA tightens its standards for rare-disease evidence.
Read more →Approvals and Regulatory Wins
The Eye Drug That Got Rejected Three Times, Then Went Over the FDA's Head
After three FDA rejections, Outlook Therapeutics' Lytenava became the first approved ophthalmic bevacizumab for wet AMD. The company won by filing a formal dispute that overturned the review division's decision. The pivotal trial showed 41% of patients gained at least 15 letters of vision versus 23% on ranibizumab. Now Outlook faces the harder challenge: selling into a market where doctors have used the cheaper, off-label version for two decades.
Read more →Clinical Data Drops
A First-in-Class Drug Targets Liver Disease by Stopping Oxidative Stress at Its Source
Calliditas reported that setanaxib, the first NOX1/NOX4 inhibitor ever tested in liver disease, hit its primary endpoint in a Phase 2b trial for primary biliary cholangitis. The higher dose cut alkaline phosphatase by roughly 19% over 24 weeks. There's virtually no competition in this drug class, but the company still needs to prove the drug actually reduces fibrosis over longer treatment periods.
Read more →Fate's Off-the-Shelf CAR-T Shows Early Signs of Life in Systemic Sclerosis
All four patients treated with Fate Therapeutics' iPSC-derived CAR-T therapy FT819 showed clinically meaningful improvement in systemic sclerosis, with zero cytokine release syndrome and zero neurotoxicity. Some were treated as outpatients. The sample is tiny, but the clean safety profile and off-the-shelf manufacturing model could give Fate an edge in the crowded autoimmune CAR-T race.
Read more →Policy and Public Health
RFK Jr. Wants a Federal Catalog of Covid Vaccine Injuries. Scientists Are Nervous.
HHS plans to publish a proposed rule in November creating a formal table of injuries presumed to be caused by Covid vaccines. A narrow, evidence-based list could help legitimately harmed patients stuck in a system that approves just 1.6% of COVID-19 countermeasure claims. But Kennedy's record of canceling mRNA research and firing CDC advisors has scientists worried the table could validate unsupported claims and further suppress vaccine demand.
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