Denmark’s Orphazyme has raised more funds to market its lead product, the heat shock protein amplifier, arimoclomol, in the US and Europe, and to evaluate the compound for other lysosomal storage and neurodegenerative disorders.
Denmark’s biotech Orphazyme A/S is raising between $83m and $97m from an offering of American Depositary Shares (ADSs) on NASDAQ in the US and a private placement in Europe, as it nears the market with its lead candidate, arimoclomol, for the ultra-orphan disease, Niemann-Pick disease Type C.
It’s the second time this year that NASDAQ Copenhagen-listed Orphazyme has raised funds to support the development and commercialization of...
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
