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.<
NICE has rejected CSL Behring’s alpha1-proteinase inhibitor Respreeza for treating genetic emphysema in draft guidance, saying that it would cost a massive £8 million to give a patient a ye