FDA Announces First Approval of Targeted Therapy Based on Basket Study
The US Food and Drug Administration (FDA) has announced that it has approved the drug vemurafenib for the treatment of patients with BRAF V600-mutant Erdheim-Chester disease (ECD). This is the first approval of a targeted therapy based on a basket study and the first-ever drug approved for ECD, a rare blood disorder. This landmark approval came as a direct result of research at Memorial Sloan Kettering Cancer Center (MSK).