FDA Grants Zelboraf Priority Review for Rare Blood Disease

Genentech has announced that the Food and Drug Administration (FDA) has accepted and granted priority review to the supplemental New Drug Application (sNDA) for Zelboraf (vemurafenib) in the treatment of Erdheim-Chester disease (ECD) with BRAF V600 mutation. ECD is a rare and serious blood disease characterized by the abnormal multiplication of histiocytes.

The application includes data from the open-label, Phase 2 VE-BASKET study, a non-randomized, basket study investigating the use of Zelboraf in patients with BRAF V600 mutation-positive cancers and other diseases, including 22 patients with ECD. Results showed that ECD patients demonstrated a best overall response rate of 54.4% as evaluated by RECIST (Response Evaluation Criteria In Solid Tumors) v1.1.

“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,” said Sandra Horning, MD, CMO and head of Global Product Development at Genentech. “[We] hope to see Zelboraf approved for Erdheim-Chester disease as soon as possible.”

The company expects the FDA to make a decision on the sNDA by December 7, 2017.

Zelboraf is already approved for the treatment of unresectable or metastatic melanoma with BRAF V600E mutation as detected by an FDA-approved test.

For more information visit Gene.com.