2024 Here we go again., page-571

Hi @Stellowe,

great point you made about the EAP adolescents. There's also the data from the RCT. A reconsideration of that data might speed the adult indication up a bit, but I still think they're going to need a dedicated adult study/ trial - perhaps observational, CIBMTR-based for approval purposes. Not just a subset analysis (arguably post hoc).

Posters should keep in mind that 'label extension' after the paediatric indication approval requires a separate submission to the FDA .

Hard to know IMO what CBER would settle for (in terms of additional efficacy evidence) for the efficacy supplement with the adult population, especially after theyve gone to the trouble of protocol discussions with BMT CTN involved.

But there's no doubt it will be IMO an Efficacy Supplemen to the paediatric indciation, when approved, viz:




Of course, post-paediatric approval. Ha. At that point, maybe anything goes.

Cheers