The company anticipates an addressable global market for progressive familial intrahepatic cholestasis of 2,500 patients, with initial plans to focus on around 100 physicians at 60 centers.
Albireo Pharma Inc. anticipates a rapid launch of Bylvay (odevixibat) after it received back-to-back approvals from the US Food and Drug Administration and European Medicines Agency for progressive familial intrahepatic cholestasis (PFIC), with reimbursement expected to come through over the course of the second half of the year in the US and Europe.
The Boston-based company said 20 July that it received FDA approval for Bylvay for pruritis in all subtypes of PFIC,...
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
