@Southoz In most instances this may be correct however, when the...

@Southoz In most instances this may be correct however, when the patient cohort is one defined as unresponsive to best available treatment and they are the patient group of whom are considered the most severe and highest risk of dying. The FDA will look at this differently, especially within the paediatric cohort, as we know RCT’s have ethical consequences hence the single arm trial and it has demonstrated the best response in terms of survival that any other medication has in such a severe grade of disease. So yes, the survival data is the cornerstone of the resubmission in conjunction with the potency assays demonstrating a relationship between drug MOI and response. This is why the FDA have performed a 10 day manufacturing inspection, have accepted the BLA resubmission and why it will be approved before or on the 2nd of august