The FDA has been busy over the past month, from expanding a gene therapy to younger patients to weighing whether “wellness” peptides belong on pharmacy compounding lists.
Here are four things to know:
1. The FDA reshuffled its peptide advisory panel to include industry advocates.
Ahead of a July 23-24 meeting on whether seven “wellness” peptides — including BPC-157 and TB-500 — should be added to the 503A Bulks List for pharmacy compounding, the FDA released a Pharmacy Compounding Advisory Committee roster where eight of 12 voting members run private wellness, longevity or regenerative medicine clinics rather than the university and federal researchers who staffed prior panels. Several members have direct financial ties to the peptide industry, including one who separately promotes BPC-157 to social media followers and charges for consultations.
The meeting comes as ECRI and the Institute for Safe Medication Practices warned that most peptide safety data comes from animal studies, that gray-market product testing has found purity as low as 5% with contamination above toxicity thresholds, and that a fatal cardiac event, case reports of melanoma and kidney dysfunction, and one death following IV administration have all been linked to the peptide class.
2. FDA expands Casgevy to children as young as 2.
The agency expanded approval of Vertex Pharmaceuticals’ gene therapy Casgevy (exagamglogene autotemcel) to patients 2 years and older with sickle cell disease with recurrent vaso-occlusive crises or transfusion-dependent β thalassemia, making it the first gene therapy approved for children this young with sickle cell disease. The one-time, CRISPR/Cas9-based treatment was previously limited to patients 12 and older. The FDA granted the supplemental approval July 1, just 53 days after filing, through the Commissioner’s National Priority Voucher pilot program.
3. An FDA panel backed the first mRNA flu vaccine.
An advisory panel recommended approval of Moderna’s mFlusiva for adults 50 to 64, and for adults 65 and older if a specific condition is met, at a June 18 meeting. The vaccine was about 27% more effective than a standard flu shot in a late-stage trial and could move from strain selection to rollout in two to three months, versus about six for traditional flu vaccines. The World Health Organization recommended updating all three flu strains for 2026 after a new A(H3N2) variant emerged that current vaccines only partially protected against, and supporters say mRNA’s shorter production window could let manufacturers match strains later in the season.
However, the recommendation lands amid continued federal skepticism of mRNA technology. HHS canceled nearly $500 million in mRNA vaccine contracts in 2025, and mFlusiva still needs full FDA approval and a CDC recommendation before reaching the market.
4. The FDA’s real-time clinical trial model is moving from concept to practice.
The agency debuted the model in April to monitor safety signals as trials are underway rather than only after they conclude, and the first trials launched with MD Anderson Cancer Center, the University of Pennsylvania, AstraZeneca, Amgen and Paradigm Health. Oncology leaders told Becker’s the shift could compress drug-development timelines — traditionally 10 to 12 years — but several flagged a risk that only sites with strong data-capture infrastructure will be able to participate, potentially widening gaps in trial access between academic and community settings.
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