The Food and Drug Administration (FDA) has approved Zelboraf (vemurafenib; Genentech) for the treatment of adults with Erdheim-Chester Disease (ECD) with BRAF V600 mutation. ECD is a rare and serious blood disease characterized by the abnormal multiplication of histiocytes.

The approval of Zelboraf for the treatment of ECD was based on data from the Phase 2 VE-BASKET study which included 22 patients with BRAF-V600-mutation positive ECD. The study measured the percent of patients who experienced a complete or partial reduction in tumor size (overall response rate). In the trial, 11 patients (50%) experienced a partial response and 1 patient (4.5%) experienced a complete response.

The most common adverse events associated with therapy included arthralgia, maculo-papular rash, alopecia, fatigue, prolonged QT interval, and papilloma. 

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Zelboraf, a kinase inhibitor, is also indicated for the treatment of unresectable melanoma or metastatic melanoma with BRAF V600E mutation as detected by an FDA-approved test.

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