A new oral inhibitor, cobimetinib (Cotellic) has been launched by the Memorial Sloan Kettering Cancer Center (MSK) for the treatment of adult patients with histolytic neoplasms (HN) (Erdheim-Chester Disease, Rosai-Dorfman, Langerhans Histiocytosis).
The treatment is intended for patients who do not harbor the BRAF V600 mutation.
Cobimetinib is an oral inhibitor of MEK1 and MEK2, currently approved to treat melanoma.
Histiocytosis is a family of blood diseases that affects both adults and children. The available treatment options for adults with HN are limited and based primarily on anecdotal case reports and poor long-term tolerance.
The breakthrough designation given by the U.S FDA was based on the result of positive findings from the phase II trial of single-agent cobimetinib for adults with histolytic disorders.
Regardless of the tumor genotype, the study enrolled 18 patients who had a histiocytic neoplasm. Those with BRAF V600 mutations were eligible only if they have intolerance or resistance to prior BRAF-targeted therapy or could not access BRAF inhibitor therapy.
For people who have histiocytosis with BRAF V600 mutations, the BRAF inhibition with vemurafenib (Zelboraf) is highly effective and is the only FDA-approved treatment for histiocytosis.