The Swiss company believes it has collected sufficient new data on Raxone for Duchenne muscular dystrophy to convince regulators and hopes to file for that indication on both sides of the Atlantic by the end of the first quarter of 2019.
Santhera Pharmaceuticals AG is still convinced that there is a future for Raxone (idebenone) as a treatment for Duchenne muscular dystrophy (DMD) despite repeated knockbacks from European regulators, as sales of the drug in its already-approved indication of Leber’s hereditary optic neuropathy (LHON) continue to climb.
The Swiss drugmaker has had a tough time to date in its talks with the European Medicines Agency with the...
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.
