Vemurafenib (Zelboraf) has been granted a supplemental new drug application and priority review by the Food and Drug Administration for the treatment of Erdheim-Chester disease with BRAF V600 mutation, according to a press release issued by Genentech.
The FDA is expected to make a decision on the indication by Dec. 7, 2017. Vemurafenib is approved for the treatment of unresectable or metastatic melanoma with BRAF V600E mutation.
The supportive data for the application came from VE-BASKET, a phase 2, nonrandomized study investigating the use of vemurafenib for people with various BRAF V600 mutation–positive cancers and other diseases. Final results for the 22 people with Erdheim-Chester disease showed a best overall response rate of 54.5% by RECIST v1.1 criteria.
The median duration of response, progression-free survival, and overall survival were not reached at a median follow-up time of 26.6 months. The most common grade 3 or higher adverse events were new skin cancers, high blood pressure, rash, and joint pain. Initial study results were published in the New England Journal of Medicine in August 2015.
Based on available published data, there are fewer than 500 cases of Erdheim-Chester disease in the United States. More than half of affected people have BRAF V600 mutation–positive disease, and there are no approved treatments, according to the release.