The Irish firm needs to give regulators in the US more information on its epidermolysis bullosa therapy but is confident of address those requests from its "existing data within the time periods required," according to CEO Joe Wiley.
Amryt Pharma's hopes of getting a thumbs-up for Filsuvez by the end of the year have hit a bit of a bump with the news that US regulators have delayed a review of the Irish firm's butterfly skin therapy by three months.
The Dublin-headquartered group had a Prescription Drug User Fee Act target action date of 30 November for Filsuvez, which is...
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
