Symdeko, which will have a wholesale price of $292,000 per year, has got the green light in the US, adding to Vertex' already-approved cystic fibrosis treatments Kalydeco and Orkambi. Now, focus will shift onto its role in potential triple combinations for the disease.
Vertex Pharmaceuticals Inc.'s domination of the cystic fibrosis market continues apace with the US Food and Drug Administration giving the nod to Symdeko, the company's third disease-modifying treatment to be approved for the rare genetic disorder.
Symdeko combines tezacaftor, which restores CFTR (cystic fibrosis transmembrane conductance regulator) gene function by moving the protein into the correct...
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
