Baldwidx
While there are welcome, positive signals coming from the new FDA Commissioner regarding rare diseases, my enthusiasm is more tempered.
We spoke to another sponsor who has been told that they may be able to file for approval this year using Phase 2 drug for a severe autoimmune disease where options are not good today.This is already a possibility under the Accelerated Approval pathway – namely, the FDA grants approval based on Phase 2 data but the drug sponsor must then conduct a Phase 3 confirmatory trial.
We are hearing an even more libertarian approach ahead with Makary describing approval pathways based on a single Phase 3 …Dare I say that it is pretty much standard already that just one Phase 3 trial is required for most rare disease drugs?
….or even no Phase 3 if there is plausible biology at work and a disease is rare enough.“If the disease is rare enough…” is the important bit here.
How rare? Very, very rare, it would seem, from what Dr. Makary has said in two separate interviews.
When you are talking about rare diseases, a genetic issue that affects 52 kids in the world and that’s a real thing…or 15 kids…, you can’t expect the companies to do a randomized controlled trial.https://www.thefdalawblog.com/2025/...or-fda-a-new-ultrarare-approval-pathway-more/….so, if a condition affects 19 people in the world as a partial triplication chromosome 15 disorder does, or a disease that affects 52 kids in the world, we cannot require two randomized control trialshttps://www.genengnews.com/topics/d...e-eyes-rare-disease-specific-review-pathways/As for whether Neuren might be allowed to run just one Phase 3 in a first indication for NNZ-2591 and then skip Phase 3 for all following indications, I doubt it.
Dr. Makary is on record as being highly critical of drug companies that he believes “game the orphan drug system” by expanding use beyond the originally approved indication.
But we can always hope for a change of mind...
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