This is an extract from a investment bank in the USA (Stifel). Put a smile on my face
What’s Going on at FDA? Cause for Optimism?
In our biopharma update report a week ago we indicated that we were hearing rumors that FDA is looking to significantly accelerate the pace of approvals in the remainder of 2025.
We emphasized that this information was tentative and based on market rumor.
This week we can confirm this rumor with a lot more specificity.
Specifically, FDA Commissioner Marty Makary indicated last week in an interview with Megyn Kelly that he would like the agency tomeaningfully accelerate drug approvals.
He noted that the case for doing this with drugs for rare disease is particularly strong.
We have heard quite strong confirmation from knowledgeable sources that senior staff at CDER and CBER are looking to “put the afterburners on” for drug approvals that meet significant unmet needs and can save lives:
- Highest priority is rare disease
- But a real sense that the FDA can do more to speed up approvals for drugs that will make a big difference for patients – e.g., in chronic diseases
- A desire to get away from bureaucratic designations that certain drugs are “breakthrough” etc. and just get to work on getting files through
We are also hearing an acknowledgement from inside sources that some applications may be quite complex, and that with personnel cuts there could be delays and higher probabilities of a CRL, but FDA will work very hard so that this does not happen.
Other knowledgeable sources indicate that Trump was put off that his “right to try” idea from the first Administration went nowhere.
For Trump 2.0, the idea is that FDA itself will take more of a “right to try” attitude. If there is a drug that could really work for a difficult cancer, rare disease or the like, perhaps the FDA could work to get the file approved quickly. Obviously, reviewers and division directors have to work with the law.
The whispers on this major shift at FDA got a lot louder last week and were shared with us by more than a few connected individuals. In addition, sponsors are having surprisingly permissive conversations since Makary has joined FDA.
- To illustrate, we spoke to one sponsor this month who had a recent meeting at FDA regarding the approval of a drug that would serve something like a thousand children with a likely fatal disease:
- The sponsor had data that the drug was making a difference in a small group of patients and that the efficacy signal was strong.
- The sponsor was going through its plans for a Phase 3 study to confirm the signal relative to historical controls.
- The FDA attendees asked why the sponsor was going to do a Phase 3 at all. Why not just file for approval?
- The conversation quickly shifted to what it would take to file this drug – mainly CMC work etc
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.
There was a sense of easing barriers for approval for rare disease drugs in the last year of the Biden Administration. We are hearing an even more libertarian approach ahead with Makary describing approval pathways based on a single Phase 3 or even no Phase 3 if there is plausible biology at work and a disease is rare enough.
We spoke to some investors about these developments last week. We see this as highly bullish for biotech in 2025.
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