FDA Approves Melanoma Drug to Treat Blood Cancers

Cotellic, an oral inhibitor of MEK1 and MEK2, was approved to treat patients with histiocytic neoplasms based on a phase 2 trial conducted solely at Memorial Sloan Kettering Cancer Center.

The FDA okayed Cotellic (cobimetinib) to treat adult patients with the family of blood diseases known as histiocytic neoplasms (HN). The diseases include Erdheim-Chester disease, Rosai-Dorfman disease, and Langerhans cell histiocytosis.

Cotellic is an oral inhibitor of MEK1 and MEK2 that currently approved to treat melanoma, Memorial Sloan Kettering Cancer Center (MSK) said in a news release. Before now, available treatment options for most adults with histiocytic neoplasms were limited and associated with poor long-term tolerance, according to MSK.

The approval was granted based on data collected by MSK in collaboration with Genentech, a member of the Roche Group, from a single-institution phase 2 trial of single-agent Cotellic for adults with histiocytic disorders. “A rarity in the world of cancer research, the clinical trial that led to the approval was conducted solely at MSK,” the organization said.

“The approval of cobimetinib represents the collective hard work of several years of investigation by many MSK researchers. There have been tremendous advances in the field of rare cancers as a result of research and trials conducted at MSK, and this approval is an excellent example of a practice-changing outcome,” said Eli L. Diamond, M.D., neuro-oncologist at MSK and principal investigator of the trial. “There has always been an unmet need for patients with histiocytosis, and we are thrilled that with this approval, these patients will now have access to a viable treatment option.”

In October 2019, the FDA announced breakthrough therapy designation to Cotellic for MEK inhibition in histiocytic neoplasms, based on a trial published in Nature by a team of MSK researchers led by Diamond; Omar Abdel-Wahab, MD, hematologic oncologist and director of the Center for Hematologic Malignancies at MSK; and David Hyman, M.D., former chief of the Early Drug Development Service at MSK.

The team used genetic sequencing test MSK-IMPACT, created by the Department of Pathology at MSK, to determine patients’ mutations.

“Until now, no standard therapy has existed for the 50% of histiocytosis patients without the BRAF V600E mutation,” said Dr. Abdel-Wahab. “The research pioneered at MSK has led to a viable treatment option for adult patients who harbor this mutation. Looking ahead, we are working on advancing treatment options for pediatric patients with histiocytosis as we have done in adults.”