Itsa, a couple weeks ago you posted link to new drafted FDA AA guidance from late 2024, I recall one big take away for me was that they are wanting the confirmatory ph 3 trial to be underway before they give accelerated approval. Sounds like FDA got tired of companies delaying these trials for years and years.
And Outlander, I've been looking into AA and similar pathways , it's interesting that on some occasions AA is not used but the therapy is approved off ph 2 data through Breakthrough Therapy Designation + Fast Track + Priority Review. As we know, technically AA is for when surrogate biomarker endpoints, or intermediate clinical endpoints, are suggesting a clinical benefit in an area of huge unmet need. As we have evidence of an actual clinical benefit with the UMSARS there could be chance FDA doesn't see the need for an AA IMO. Rather they see the most appropriate pathway as a conditional full approval using BTD +FT+ PR with the need to run further studies to confirm effect/safety and best dosing, or even no ph 3 as Stamler hints in * article. Stamler also talks on this subject at 47:50 in the results webinar.
I will add that after looking though some examples, it all does seem a little complicated to me.. with some other accelerated approvals based off clinical benefit not surrogate, which is a tad confusing. Perhaps it doesn't matter so much what pathway, perhaps FDA takes a full look at all data available and makes a call on which mechanism is most appropriate to get it to patient in need earliest, and this can sometime vary for similar scenarios. I haven't heard Stamler use the exact term "Accelerated Approval', rather talking about accelerating or fast-tracking regulatory approval, so perhaps leaving door open to push for the Breakthrough Therapy route. Just all my own opinions trying to make sense of it, happy to educated on it. cheers
This isn't the one you posted Itsa but goes into AA. The one you posted did mention the need to begin ph 3 before AA will be granted. https://www.fda.gov/media/184120/download
ATH Price at posting:
1.0¢ Sentiment: Buy Disclosure: Held
A personalised tool to help users track selected stocks. Delivering real-time notifications on price updates, announcements, and performance stats on each to help make informed investment decisions.
(20min delay)