Santhera had a tumultuous 2017 and recovery is not going to be easy this year as its lead compound for the rare disease Duchenne muscular dystrophy is about to be rejected by regulators in Europe for a second time.
By the end of last year Santhera Pharmaceuticals AG's stock price had lost more than half its value; it seems 2018 will not be kinder as the company's hopes of securing regulatory approval for a novel treatment for muscular dystrophy are set to be dashed again.
The European Medicines Agency's committee for medicinal products for human use (CHMP) will return a negative opinion for Santhera's Raxone...
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.
