The US Food and Drug Administration (FDA) has approved Genentech’s Zelboraf (vemurafenib) for the treatment of Erdheim-Chester disease (ECD) with BRAF V600 mutation.
This rare, serious blood disease is characterised by the abnormal multiplication of certain white blood cells called histiocytes, which can invade normal tissues and organs in the body.
FDA approved the treatment based on data from the Phase II VE-BASKET study carried out in 22 people with ECD.
The study showed a best overall response rate of 54.5% and the most common Grade 3 or higher adverse events were found to be new skin cancers, high blood pressure, rash and joint pain.
Genentech Global Product Development chief medical officer and head Sandra Horning: “This FDA decision means people living with Erdheim-Chester disease will now, for the first time, have an FDA-approved treatment option.
“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.”
In 2011, Zelboraf monotherapy was approved to treat people with unresectable or metastatic melanoma with BRAF V600E mutation.
Previously, the FDA granted Priority Review and Breakthrough Therapy Designation to Zelboraf for ECD with BRAF V600 mutation.
ECD is an extremely rare non-Langerhans cell histiocytosis and its exact prevalence and incidence are difficult to ascertain, given it is so rare.
Separately, the agency has approved Genentech’s supplemental new drug application (sNDA) for Alecensa (alectinib) for the treatment of people with anaplastic lymphoma kinase (ALK)-positive metastatic non-small-cell lung cancer (NSCLC).