The findings may make it possible to identify a subset of patients who experience poorer outcomes or more serious side effects, especially neurotoxicity.
In a CRL issued for valoctocogene roxaparvovec, the FDA told BioMarin that differences between its Phase I/II and Phase III studies limited the ability to rely on the earlier trial to support durability of effect, which an analyst said likely referred to a product comparability… Read More »FDA rejects BioMarin’s hemophilia A gene therapy in ‘massive surprise’
The company said the study of Kymriah in follicular lymphoma met its primary endpoint, though it did not disclose the trial data. It plans to submit approval applications to the FDA and EMA next year.
The FDA approved Monjuvi for second-line diffuse large B-cell lymphoma. Though the drug has the same antigen target as approved CAR-T cell therapies, it is designed for patients unable to tolerate such intensive treatments.
By CHADI NABHAN, MD, MBA, FACP “The goal for me and for my clinical and research colleagues is to put ourselves out of a job as quickly as possible”. This is how Mikkael Sekeres ends his book “When Blood Breaks Down” based on true stories… Read More »“When Blood Breaks Down”: It Can Break Your Heart