Govorestat could soon be filed for a second indication after impressing as a potential treatment for SORD deficiency, one of the most common forms of recessive hereditary neuropathy for which there are no approved therapies.
Having recently submitted govorestat on both sides of the Atlantic for galactosemia, Applied Therapeutics Inc. will be knocking on the regulators' doors again soon on the back of more impressive data, this time evaluating the drug for sorbitol dehydrogenase (SORD) deficiency.
The New York-headquartered biotech has unveiled positive interim 12-month results from the ongoing Phase III INSPIRE trial evaluating once-daily oral...
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.
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.
