Sobi is paying big bucks to tap into the potential of Apellis's pegcetacoplan, a targeted C3 therapy for the treatment of multiple rare diseases with high unmet need which the partners say impact more than 275,000 patients globally.
Swedish Orphan Biovitrum AB is pressing on with plans to diversify the pipeline beyond its core hemophilia franchise and has licensed rights to Apellis Pharmaceuticals, Inc.'s pegcetacoplan, a targeted C3 therapy for the treatment of multiple rare diseases including paroxysmal nocturnal hemoglobinuria (PNH).
The complex pact will see Sobi pay an upfront fee of $250m plus $80m in development reimbursement to Apellis and...
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.
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.
