That is the question. Not even Z or Kontro can give you an exhaustive answer. The FDA mentions it as an endpoint in the draft, but that doesn’t mean yet that achieving it leads to marketing authorization. The problem with CR is that if those who received a transplant are controlled for, the significance is diluted. In other words, it is a confounding factor.
If Faron tries Phase 3 with CR as the primary endpoint, an SPA (Special Protocol Assessment) would likely be appropriate. Otherwise, the regulatory risk is too high—meaning that in the end, they might be told it was a great trial, but it isn’t sufficient evidence for marketing authorization.
But this whole discussion is premature, as the start of Phase 3 will likely be in 2028 at the earliest. First, Phase 2b must be completed.
If it is not an endpoint, it cannot be used for labeling—that is, what the drug is claimed to do. And that matters for how much can be charged for the drug $ (or what people are willing to pay).