The last eighteen months have been an unprecedented time of unpredictability for the FDA, but moving forward we’re likely going to see an emerging consistency: a higher amount of FDA flexibility on rare diseases.
It’s already begun and is likely to accelerate with Prasad and Makary gone.
How is this manifesting?
FDA flexes flexibility
I wrote last week about UniQure getting some friendly treatment from the FDA. Now there’s this: Wagering on FDA changes, Regenxbio will submit Duchenne gene therapy for approval.
Early on in this administration the FDA signaled more flexibility. However, the highly publicized rollout of that end up all over the place. Some applications seemed to get harsh review at the agency, while other arguably similar ones got the flexibility they wanted.
With that backdrop, my guess is that the FDA is going to be erring on the side of much more flexibility. In some cases, too much. Who’s going to say “no” these days?
More recommended reads
- Derivation of elephant induced pluripotent stem cells, Nat Methods. I wrote before about Colossal’s preprint on elephant iPS cell generation. The worry here is that this biotech will claim in 5-10 years to have de-extincted woolly mammoths, when they almost certainly won’t have achieved that. What’s likely though in my view is that they will have put elephants at risk.
- Cynata’s Cymerus flops in osteoarthritis, GVHD, BioWorld. Harsh headline.
- No Evidence for Trump’s Right to Try Claim, Fact Check. People often invoke RTT for unproven stem cells and there is that national RTT law. Sponsors seem relatively uninterested in participating. Expanded access is an efficient, popular alternative.
- Digit regeneration in mice is stimulated by sequential treatment with FGF2 and BMP2, Nat. Comm. Wouldn’t it be exciting if we could regrow digits? Want to learn more? Read about blastemas.
Base editing of human embryos
Here’s my latest STAT column sparked in part by Dieter Egli’s preprint on efficient, specific base editing of human embryos. New human embryo editing advances require tough conversations on ethical boundaries.
Now a new Nature paper from Kathy Niakan reports on base editing of human embryos more squarely focused on basic research questions: Base editing reveals an essential role for NANOG in human embryogenesis. From the pub: “Retention of primitive endoderm differentiation in NANOG-edited human embryos reveals a functional compensation distinct from mouse, underscoring the importance of directly investigating human development.”
More exciting work of this type is sure to be coming.
Some are likely to want to misuse this technology though for reproductive purposes.