Roche’s Hemlibra has been approved in an expanded use in haemophilia A in the US, as the Swiss firm seeks to build a new blockbuster franchise in rare diseases.
As uncertainty hangs over the availability of Vertex’s cystic fibrosis drug in England, the company has signed an agreement that gives Danish patients access to Orkambi.
Alnylam may have to put hold off plans for an early FDA approval for its gene-silencing drug givosiran – because recruitment to a rare disease trial is going better than expected.<
A pharma company’s core mission is to improve patient outcomes. This hinges on effectively influencing HCP clinical behaviour and driving disease education.