Even with numerous endpoint misses, such is the demand for treatment options for the ultra-rare disease Niemann Pick, Orphazyme is pushing forward with its US and European regulatory filings of arimiclomol, which has no approved therapies in the US and one in the EU.
Danish rare disease company Orphazyme AS is preparing to file its lead candidate arimoclomol for Niemann Pick disease type c (NPc) with the regulators based on data from its Phase III study, despite it not showing significance on primary or secondary endpoints.
The 12-month study, which recruited 50 patients aged 2-18, was a double-blinded, placebo-controlled interventional study that aimed to show efficacy...
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
