Amicus Pompe Disease Combo’s Approvability In Question As Key Trial Misses Mark
Company Touts ‘Totality Of Data’ As Supporting Approval
The company hopes to score a nod from the US FDA based on a secondary endpoint that won approval for Sanofi’s Lumizyme.
You may also be interested in...
Other antibody treatments have shown efficacy in early-stage disease after faltering in hospitalized patients.
The company plans to start its Phase III trial of SRP-9001 in the middle of this year, with a potential readout by the end of 2022.
The RNA drug’s accelerated approval brings Sarepta’s exon-skipping market share to nearly 30%.