In addition to the ODAC vote and successful P3 trial (and we know for a fact that the FDA has come to understand that single arm trials are ok for this condition, per its own draft industry guidance on “a” and cGVHD put out around Aug/Sep last year), additional long term aGVHD survival data were provided last year in the resubmission in order to support long term clinical benefit in the real world. At least one of the company’s market releases mentioned that for paediatric approval, satisfactory potency assay data OR an adult trial would be required.
I don’t think there is much ambiguity at all. What the company keeps emphasising also, is the commercial benefit behind the proposed adult trial. (It may, of course, also be trying to deflect criticisms that it didn’t do the adult trial earlier, and ended up wasting valuable time and copping so much flak, but now that the adult trial is not going to cost much because of the backing from the BMT-CTN, I think you can equally argue that it is a monetary saving to the company that might not have been available had the decision to go ahead been made a few years ago.)
Could AA have been discussed explicitly by the FDA for heart? Absolutely, as @ddwn mentioned above. There was indeed something like a surrogate endpoint in the LVAD trial, being the reduction in GI bleeding which was statistically significant, and if the FDA is willing to treat the DREAM trial as a confirmatory trial for the LVAD trial, with another small, focused follow-up study of a population already known to benefit more from the therapy, then it’s a fantastic outcome. The LVAD indication already has got an RMAT designation, which admits the use of surrogate endpoints in considering an AA.
So I actually thank the confounding questions from the naysayers.
MSB Price at posting:
31.5¢ Sentiment: Hold Disclosure: Held
(20min delay)