The NDA submission is supported by data from a multicentre phase 3 RCT (n=217) which found that 59% of the veverimer group vs 22% of the placebo group achieved the composite primary efficacy endpoint, either a normal serum bicarbonate concentration or increase by 4 mmol/L (p<0.0001).
In addition, a 40-week long term safety and efficacy extension study (n=196) reported that at week 52, more patients on veverimer vs placebo had an increase in bicarbonate (≥4 mmol/L or normalisation, 63% vs 38%, p=0.0015) and higher bicarbonate concentrations were observed at all time-points starting at week 1 (p<0.001).