A big year for sickle cell

The approval of Agios’s mitapivat this month in the rare disease pyruvate kinase deficiency highlighted the tricky pricing decision the group must take should the same compound be approved in the more common condition sickle cell anaemia.

Several other sickle cell agents are also approaching the market: the filing of Crispr Therapeutics’ gene-edited CTX001 is imminent, and two more candidates have recently entered late-stage trials. A competitive price could be crucial for Agios – though some industry experts have suggested that the group might simply shelve the project in this indication, despite it having just started a phase 2/3 study here.