Wow Hottod - this is remarkable if true and should surely bode well for us.
Re the link you provided above, was particularly taken by some of the following comments (highlights my own):
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.
This really is remarkable if accurate. I know I am repeating myself, but I can hardly believe it. If it is accurate, I think it completely reshapes our company/value. We obviously knew of the pipeline in a drug approach and knew that there is significant upside/value in a drug that treats multiple conditions, but the challenge is always getting through regulatory burdens and doing so in a timely and cost-effective manner. The process just eats away at the profit margin and subsequent value proposition - thereby lowering our acquisition value.
This revised approach (if accurate) turns that on its head completely in my view.
It very well could place us in the position whereby we may be able to run one P3 in PMS as the cornerstone (proof of concept for the drug) for significantly expedited future approvals in other conditions.
It may be the case that one successful P3 trial serves to establish the efficacy of the drug over placebo, validates its safety profile and then moving forward, you may only need to conduct P2 trials in new conditions (given the established safety profile) and then seek direct approval as per the commentary above.
This is outside my field of expertise but that would surely materially change/extend the peak sales timeline for NNZ 2591 if the FDA adopts this approach. You could conceivably get the drug to market 18-24 months earlier than previously planned, which is extraordinarily valuable in one condition, let alone the current 4, with potentially more to follow. Your CDF calculations and valuations change materially!!
My mind is blown! I have asked about this previously on the forum re whether success in one P3 could be leveraged in other conditions/trials and skip them all together and the general consensus is that its not how the FDA works - but that may not be the case moving forward if the above commentary is accurate.
I read another post on the DXB thread with similar comments about the FDA's revised/expedited approach to Rare/Orphan conditions and there was a comment made about how this revised focus will drive increased interest in the Rare/Orphan space.
If that is true, and the above comments about a revised FDA approach are accurate, then it goes without saying (in my view) that this very much lifts the value of our company, specifically 2591.
Add this revised approach to the increased focus on Autism and we might find ourselves in the middle and beneficiaries of the perfect storm!
(20min delay)