ScripPTC Therapeutics expects to reach blockbuster sales for its oral phenylketonuria (PKU) drug Sephience (sepiapterin), which obtained US Food and Drug Administration approval on July 28. The firm will t
ScripData from the Phase III GENEr8-1 trial testing BioMarin’s Roctavian in patients with severe hemophilia A over a period of five years showed that the gene therapy maintained durable bleed control and s
ScripPfizer stands to expand the addressable market for its hemophilia A and B drug Hympavzi (marstacimab-hncq) with Phase III data showing it is effective in patients with clotting factor inhibitors along
Pink SheetJuly is inextricably linked with Independence Day in the US, but the calendar of applications that are up for Food and Drug Administration actions in July 2025 is notable for the number of novel candi