FDA grants priority review to Zelboraf for BRAF V600-mutated Erdheim-Chester disease

The FDA granted priority review to vemurafenib to treat patients with BRAF V600-mutated Erdheim-Chester disease, according to the drug’s manufacturer.

The agency also granted breakthrough therapy designation to vemurafenib (Zelboraf; Genentech) for the same indication.

Erdheim-Chester disease is a rare blood disease characterized by the abnormal multiplication of certain white blood cells. There are no FDA-approved treatments for this condition.

The supplemental new drug application included data from the open-label, phase 2 VE-BASKET study, designed to evaluate vemurafenib — a selective oral inhibitor of the BRAF V600 kinase — in patients with BRAF V600 mutation-positive cancers and other diseases.

After median follow-up of 26.6 months, researchers reported a 54.5% overall response rate among the 22 patients with Erdheim-Chester disease.

Median duration of response, PFS and OS had not been reached.

The most common adverse events included joint pain, rash, hair loss, change in heart rhythm, fatigue, skin tags, diarrhea and thickening of the skin. The most common severe adverse events included new skin cancers, high blood pressure, rash and joint pain.

“This first potential treatment submitted to the FDA for this rare disease was identified by an innovative trial studying Zelboraf across different diseases with the same genetic mutation,” 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 bringing new treatment options to people with rare diseases and hope to see Zelboraf approved for Erdheim-Chester disease as soon as possible.”

The FDA set an action date for Dec. 7.

Vemurafenib already is approved for treatment of patients with BRAF V600E mutation-positive unresectable or metastatic melanoma.