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FDA approves Zelboraf for BRAF V600-mutated Erdheim-Chester disease
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The FDA expanded approval of vemurafenib to include certain adults with BRAF V600-mutated Erdheim-Chester disease.
Vemurafenib (Zelboraf, Genentech), a selective oral inhibitor of the BRAF V600 kinase, is the first FDA-approved treatment for Erdheim-Chester disease a rare blood disease characterized by the abnormal multiplication of histiocytes, a type of white blood cell. Excess histiocytes can create tumors that infiltrate organs and tissues, including the heart, lungs, brain and others.
An estimated 600 to 700 patients worldwide have Erdheim-Chester disease, and 54% of them harbor BRAF V600E mutation.
The FDA previously approved vemurafenib to treat patients with BRAF V600E mutation-positive unresectable or metastatic melanoma.
“Today’s approval of Zelboraf for patients with Erdheim-Chester disease demonstrates how we can apply knowledge of the underlying genetic characteristics of certain malignancies to other cancers,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, said in a press release. “This product was first approved in 2011 to treat certain patients with melanoma that harbor the BRAF V600E mutation, and we are now bringing the therapy to patients with a rare cancer with no approved therapies.”
The agency’s decision included data from the open-label, phase 2 VE-BASKET study, designed to evaluate vemurafenib in 22 patients with BRAF-V600-mutation positive Erdheim-Chester disease.
Overall response rate served as the primary endpoint.
Eleven patients (50%) experienced partial response and one patient (4.5%) had a complete response.
Common side effects included arthralgia, maculo-papular rash, alopecia, fatigue, prolonged QT interval and papilloma. Severe adverse events included the development of new cancers, growth of tumors in patients with BRAF wild-type melanoma, hypersensitivity reactions, severe skin reactions, heart abnormalities, liver damage, photosensitivity, severe reactions in the eye, immune reactions after receiving radiation treatment, kidney failure and thickening of tissue in the hands and feet.
“This FDA decision means people living with Erdheim-Chester disease will now, for the first time, have an FDA-approved treatment option,” Sandra Horning, MD, chief medical officer and head of Global Product Development at Genentech, said in a company-issued press release. “We are committed to finding new ways to bring medicines to patients with high unmet need, and we are pleased that this innovative clinical trial helped identify Zelboraf for treatment of this rare disease.”