RE:RE:its important to note thatTo Kenlu1: only about 11% of rejected BLA are CMC issues (Chemistry, Manufacturing and Controls).
CMC includes manufacturing but only a part of CMC rejection is due to manufacturing.
BLA's rejection reason
1-Efficacy deficiencies only: 31.8%
2-Safety and efficacy deficiencies: 27.2%
3-Safety deficiencies only: 25.8%
4-CMC alone: 11.3%
5-Labeling alone: 2.6%
6-CMC and labeling: 1.3%
Of the unsuccessful first-time applications, around (15.9%) included uncertainties related to dose selection, (13.2%) choice of study end points that failed to adequately reflect a clinically meaningful effect, (13.2%) inconsistent results when different end points were tested, (11.3%) inconsistent results when different trials or study sites were compared, and (13.2%) poor efficacy when compared with the standard of care. This breaks downs of #1 and 2 includes also some safety concerns.
These statistic are based on a 10 year period. These number can change overtime.
For Plasminogene, walk in the park IMHO