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NICE has rejected CSL Behring’s alpha1-proteinase inhibitor Respreeza for treating genetic emphysema in draft guidance, saying that it would cost a massive £8 million to give a patient a ye
Alexion is preparing filings in major markets for its rare disease drug Soliris in a new indication after a positive readout in a rare neurological disease.
Spark Therapeutics’ and Novartis Luxturna gene therapy for a rare inherited eye disease has been okayed by European regulators, paving the way for an EU licence in the coming months.
A pharma company’s core mission is to improve patient outcomes. This hinges on effectively influencing HCP clinical behaviour and driving disease education.