The goal would be to obtain breakthrough designation status for...

The goal would be to obtain breakthrough designation status for the CPACS all comers trial, which is the fastest of the fast track approvals.


source: https://link.springer.com/article/10.1007/s10198-023-01639-x


When can breakthrough designation be granted?

As part of a pre-IND application or active IND, albeit clinical data is required to support the application.

BTD readings: https://www.fda.gov/regulatory-information/food-and-drug-administration-safety-and-innovation-act-fdasia/fact-sheet-breakthrough-therapies


details of BTD approvals

https://www.fda.gov/media/95302/download

Given FDA project Optimus there is a focus on optimised dosing / safety rather than the legacy approach of a maximum tolerated dose. This bodes well for BTD status for CPACS trial.

So when will BTD be submitted?

I think we will need to see data from P2 CPACS all comers trial before an application is submitted to support BTD. This aligns with the approach taken by Cosela, which relied on P2 efficacy data. It doesn’t appear to be common for FDA to approve based on P1 safety data alone but happy to be corrected.



This is important as it’s sets a precedence from which to gauge a timelines.

The most important point that may be overlooked, is that clinical validation, geographical licensing and partnering may occur earlier than approval. The most critical link in the chain is RC220 cGMP tox studies green light then P1 readout that will validate CPACS + FTO has translated into the clinic.

DYOR