FDA approves first Erdheim-Chester disease therapy
medwireNews: The US Food and Drug Administration has granted approval for the use of vemurafenib in Erdheim-Chester disease (ECD) in patients with a BRAF V600 mutation, the first treatment for this rare blood cancer.
The B-Raf enzyme inhibitor was previously approved for the treatment of late-stage melanoma patients with the BRAF V600 mutation.
“Today’s approval of [vemurafenib] for patients with ECD demonstrates how we can apply knowledge of the underlying genetic characteristics of certain malignancies to other cancers,” commented Richard Pazdur, from the FDA Oncology Center of Excellence, in a press release.
Around half of ECD patients carry this mutation and in an open-label trial, 11 of 22 such participants given vemurafenib achieved a partial response to treatment and one patient a complete response, with median duration of response unreached after a median of 26.6 months’ follow-up.
Vemurafenib is recommended at a dose of 960 mg twice daily taken with or without food. Over 50% of trial participants experienced adverse reactions including arthralgia, rash, alopecia, fatigue, prolonged QT on electrocardiogram, and skin papilloma. A third of patients discontinued treatment because of side effects.
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