Agios’ Mitapivat Shows Improvement In Regularly Transfused PKD Patients
Results Follow ACTIVATE Study Data In December
While representing a smaller population, the ACTIVATE-T trial targeted patients seen as having greater unmet need.
You may also be interested in...
Agios’ cellular metabolism expertise first delivered cancer drugs but sees greater opportunity in genetically defined diseases. CEO Jackie Fouse talked to Scrip about the business strategy.
Strong top-line efficacy in pyruvate kinase deficiency patients who don’t receive regular transfusions looks approvable, analysts say, but Agios will await data in regularly transfused patients.
The RNA drug’s accelerated approval brings Sarepta’s exon-skipping market share to nearly 30%.