Switching patients from the very expensive ultra rare disorder drug to ravulizumab will be a key strategy for Alexion if the latter is approved. Recent compelling data in paroxysmal nocturnal hemoglobinuria patients suggests the long-acting C5 complement inhibitor could offer more benefit than its predecessor with more convenient dosing.
Having just presented more strong data for ravulizumab, also known as ALXN1210, Alexion Pharmaceuticals Inc. is cashing in a priority review voucher (PRV) to get a faster assessment in the US for the successor to its blockbuster Soliris (eculizumab) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).
The Biologics License Application to the FDA for the investigational long-acting C5 complement inhibitor has been made with a rare...
A court order encompassing funding, drug pricing, clinical trials and overall policy implementation aspects is expected to bring about a paradigm shift in the treatment of rare diseases in India. Sarepta, Roche and Sanofi are among the key players that have been part of pricing discussions.
Merck & Co. won European Commission approval for Winrevair on top of other medicines for pulmonary arterial hypertension and expects an initial EU launch in Germany.
The company, in its new profitable era, is bringing in new leadership, with Amgen VP of medical affairs for rare diseases Greg Friberg and recently retired Roche Partnering head James Sabry.
Bluebird, Vertex and Sarepta are starting to see some commercial gene therapy traction and expect momentum to pick up in the second half, while hemophilia remains a challenging area.
Lilly's first Phase III triplet win opens the door to a label expansion in BTK-pretreated CLL, with analysts suggesting Jaypica could attain blockbuster status. However, the window to capitalize may be brief as next-generation BTK degraders close in with head-to-head trials.
Mounjaro, Eliquis, and Darzalex buck the Q1 trend to make gains
