Clinical TrialsFunctional improvement from baseline on clinical endpoints was observed with nomlabofusp treatment out to one year, which is divergent from FA-COMS natural history and has not been observed before in an FA interventional trial.
EarningsPalynziq has experienced consistent sales growth since launch, generating ~$87M in annual sales in its first full year of launch, at a ~$400M run rate.
Regulatory PathwayA written correspondence from the FDA provides a clearer path toward achieving the goal of filing under an accelerated approval pathway.