Following the failure of PledOx, Pledpharma is joining forces with Rare Thyroid Therapeutics to target orphan treatments, initially focusing on the latter's investigational therapy for MCT8 deficiency.
Plagued by clinical setbacks with its lead asset PledOx for chemotherapy-induced peripheral neuropathy, PledPharma AB has opted to focus on the orphan drug segment and acquire fellow Swedish firm Rare Thyroid Therapeutics and its ultra-orphan asset Emcitate.
The deal will see Pledpharma pay privately held RTT SEK60m ($6.7m) in cash and 63.7 million newly issued shares, giving...
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.
As clinical trials grow more complex, execution pressures are changing where and how evidence is collected.
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.
