MSB to dispute FDA finding in Type A meeting, page-364

agreed. Really agree with all of what kervio said, but the key point here is we are looking at a clinical unmet medical need for children with a relatively high mortality rate. If this were not an unment medical need (i.e. there were already options available for these children) then id say definitely FDA will be staunch on their opinion, but we have a good case with nothing else on the market (acellerated approval conditional with adult RCT) AND its completely safe (basically patients have nothing to lose) and manufacturing seems to be fine.