The company got FDA approval for the drug in Bardet-Biedl syndrome, on top of the existing approval from November 2020 in obesity due to POMC, PCSK1 and LEPR deficiency.
Having taken the “pipeline in a product” approach to developing Imcivree (setmelanotide) in rare diseases of obesity is enabling Rhythm Pharmaceuticals, Inc. to leverage its existing commercial experience as a launchpad for Imcivree’s latest approved indication.
Rhythm received US Food and Drug Administration approval for Imcivree in monogenic or syndromic obesity due to Bardet-Biedl syndrome (BBS)...
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
