FDA Approves Cobimetinib for Histiocytic Neoplasms

Patients with histiocytic neoplasms can now receive treatment with cobimetinib following its approval by the FDA.

Oral MEK1/2 inhibitor cobimetinib (Cotellic) has received approval by the FDA for the treatment of patients with histiocytic neoplasms including Erdheim-Chester disease, Rosai-Dorfman disease, and Langerhans cell histiocytosis.

The approval was based on findings from a single-institution phase 2 trial (NCT02649972) conducted by Memorial Sloan Kettering Cancer Center (MSKCC) and Genentech in a population of adult patients with histiocytic disorders.

“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 MSKCC, and this approval is an excellent example of a practice-changing outcome,” principal investigator Eli Diamond, MD, neuro-oncologist, and neurologist at MSKCC, said in the press release. “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.”


FDA approves oral MEK inhibitor cobimetinib for histiocytic neoplasms, research led by Memorial Sloan Kettering Cancer Center. News release Memorial Sloan Kettering Cancer Center. November 1, 2022. Accessed November 2, 2022. http://bit.ly/3Dn4Lwk