Positive results for the Swiss major's oral therapy suggests that Biogen and Ionis' monopoly of the lucrative spinal muscular atrophy market with Spinraza could soon be under considerable threat.
A few weeks after stopping the development of its spinal muscular atrophy (SMA) candidate olesoxime, Roche has presented successful data from a trial of risdiplam, now its most advanced compound to tackle a disorder that is the leading genetic cause of mortality in infants and toddlers.
The Swiss major and partners PTC Therapeutics Inc. and the SMA Foundation in the US have presented an update...
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
