Actelion spin-off Idorsia is becoming the largest shareholder in Santhera after deciding its fellow Switzerland-based group is best placed to advance its optioned DMD candidate vamorolone.
With one Duchenne muscular dystrophy (DMD) candidate already on its hands in Raxone (idebenone), Santhera is close to getting hold of another one - vamorolone - through a option deal inked with fellow Swiss group Idorsia.
Santhera has bought the rights to sub-license vamorolone, which was discovered and is being developed by Reveragen BioPharma Inc....
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.
