FDA Gives Approval to Zelboraf, New Drug for Treating Erdheim-Chester Disease

Published Date : Nov 07, 2017

The U.S. Food and Drug Administration (FDA) has given a nod to a new drug, Zelboraf (vemurafenib), for treating Erdheim-Chester Disease (ECD). Formerly, the drug had been approved for BRAF V600E, a genetic mutation. However, it has now been shown the green light for ECD as well. It is the supposedly the first drug approved for this rare type of blood cancer. The approval, according to FDA’s Oncology Center of Excellence director, Richard Pazdur, M.D., has showcased how doctors could use the knowledge of the latent genetic characteristics of certain malignancies to other types of cancer.

An FDA statement revealed that the new drug is a kinase inhibitor capable of terminating growth in certain cells by hindering key enzymes.

Priority Review and Orphan Drug Status for Zelboraf

Zelboraf had been tested on 22 ECD patients having the BRAF-V600-mutation. While only one patient demonstrated a complete response for complete tumor reduction, 11 of them showcased a partial response, giving an evidence of the efficacy of the drug. Manufacturers had to submit the results of their clinical trials that showed the safety and effectiveness of the drug to receive an approval.

A safety trial unveiled that the drug could lead to change in the heart’s electrical activity, hair loss, and joint pain. The development of secondary cancers could also be on the cards. Furthermore, it could cause allergic reactions such as Stevens-Johnson Syndrome (SJS) but rarely lead to severe liver damage. Pregnant women have been advised to abstain from using the drug.