FDA review of the company's BLA for a hemophilia gene therapy could be delayed by three months to allow the agency to review a three-year data analysis from an ongoing trial.
BioMarin Pharmaceutical Inc.'s biologics license application (BLA) for the hemophilia A gene therapy valoctocogene roxaparvovec could face another delay to allow the US Food and Drug Administration more time to review additional data from a three-year analysis from the ongoing Phase III GENEr8-1 study.
Even as BioMarin is ramping up the launch of the gene therapy in Europe, where it was granted conditional approval...
More than 100 biopharma executives and experts told Scrip their predictions for therapeutic area advances in the coming year. The recent commercial success of GLP-1s in diabetes and obesity and their potential in further disease areas fuelled excitement around the metabolic space. Expectations were also positive in neurology following the launch of Leqembi for Alzheimer’s disease in 2023, while the multiple opportunities to improve cancer treatment kept oncology top of the pile overall.
In this week's podcast edition of Five Must-Know Things: Merck & Co. steps into CD19 bispecific space; gene therapy patients rise, but slowly; Madrigal’s Rezdiffra plans; Korean biopharma financing recovering?; and approvals to watch out for in Q3.
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.
After 10 years of promised investment following its Nobel Prize for iPS cell research, Japan is cautiously narrowing regulations around the conditional approval of cell therapies and cutting some reimbursement prices. Commercial success remains mixed and some products have been withdrawn from the market.
