FDA rejects BioMarin’s hemophilia A gene therapy in ‘massive surprise’

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 issue.

FDA approves MorphoSys, Incyte’s drug for vulnerable lymphoma population

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.

“When Blood Breaks Down”: It Can Break Your Heart

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 of patients with leukemia. I share Mikkael’s sentiments and have …

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