Clinical TrialsThe phase 3 SCD trial mirrors the phase 2 trial, providing familiarity with patient population and endpoints, which is seen as a positive aspect.
Market PotentialMitapivat demonstrated significant reductions in VOCs compared to placebo, with strong potential for success on both endpoints of Hb improvement and VOC reductions in Phase 3.
Regulatory MilestonesApproval in Saudi Arabia and a positive CHMP opinion for thalassemia subtypes support confidence in upcoming regulatory milestones.