The commercial team is on board and ready to market the first FDA-approved RNAi therapeutic patisiran for hereditary transthyretin-mediated amyloidosis.
Alnylam Pharmaceuticals Inc. is offering outcomes-based contracts for its small interfering RNA (siRNA) therapeutic Onpattro in the rare disease of hereditary transthyretin-mediated amyloidosis. The drug, which has an annual list price of $450,000, was shipped to customers within 48 hours of US FDA approval on Aug. 10.
FDA approval was a breakthrough in a number of respects, as Onpattro (patisiran) is the first RNAi therapeutic approved in...
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.
