Share Price, page-3045

Spot on KB. Once the trials are 'open label' the diarrhoea becomes just that, for better or for worse - and no longer a possible unblinding signifier. Hopefully the open label trials are showing at least ongoing continued symptom relief for girls from the Trof arm and (very importantly) symptom improvement from those on the placebo arm. After all, that is the what will finally make or break this drug (if approved).

However, blinded placebo based two arm trials are done for a reason and the integrity of the blinding is of paramount importance . Patients/doctors/caregivers must be unaware of which arm is involved, otherwise the intention of the blinding is compromised.

Did the diarrhoea compromise the blinding? IMO, that is not a matter of trial data, but of subjective opinion. No amount of additional trial data can assist here. It is the opinion of the FDA that matters.