FDA expands approval of Roche's Zelboraf to treat certain patients with Erdheim-Chester disease

Roche's Genentech unit on Monday announced that the FDA approved Zelboraf (vemurafenib) for use in patients with Erdheim-Chester disease (ECD) who carry the BRAF V600 mutation. Richard Pazdur, acting director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research, said "today's approval of Zelboraf for patients with ECD demonstrates how we can apply knowledge of the underlying genetic characteristics of certain malignancies to other cancers." 

Approval for the expanded indication was supported by findings from the Phase II VE-BASKET trial, which included 22 patients with BRAF V600 mutation-positive ECD. According to the FDA, study data showed that the therapy was associated with a best overall response rate of 54.5 percent, consisting of 11 partial responses and one complete response. 

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In August, the FDA awarded priority review and breakthrough therapy designation to Zelboraf for the treatment of BRAF V600 mutation-positive ECD. The agency also granted Zelboraf orphan drug status for this indication. 

Zelboraf was initially authorised by the FDA for the treatment of certain patients with metastatic melanoma. The BRAF inhibitor was jointly developed with Daiichi Sankyo under the terms of a 2006 agreement. 

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