The Evolving Treatment Landscape for Myelofibrosis

John Mascarenhas, MD, professor of medicine at Icahn School of Medicine at Mount Sinai, director of the Center of Excellence for Blood Cancers and Myeloid Disorders, and a member of The Tisch Cancer Institute, summarizes the treatment landscape for myelofibrosis, which now includes of pacritinib (Vonjo).

Prior to the FDA approval of pacritinib, the available JAK inhibitors for the treatment of myelofibrosis were ruxolitinib (Jakafi) and fedratinib (Inrebic). Both agents were able to improve spleen symptoms in patients, but there was an unmet need for an option to treat patients with low platelets, according to Mascarenhas.

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0:08 | The treatment for myelofibrosis has first and foremost been the use of a JAK inhibitor to try to address mainly spleen and symptom burden. Ruxolitinib was approved in 2011 based on the COMFORT studies and then fedratinib was approved in 2019, based on the JAKARTA and JAKARTA-2 studies, and is mostly relegated to the second-line setting and these 2 drugs have been a big step forward in improving symptoms and spleen burden for patients. However, their uses are relegated to those with platelets above 50,000, and that represents an unmet need for those patients with myelofibrosis and thrombocytopenia, I would argue [treating platelet counts] less than 100,000, but specifically less than 50,000, is an unmet need in which we do not have effective therapies that we can be delivered in a safe manner that can afford this same type of benefit that those patients who have higher platelets.

1:05 | We know that patients with myelofibrosis that have low platelets have poor survival outcomes. There are limited treatment options, and these patients are in need of therapy. So, that really left a window open for the final development and approval of pacritinib, which I'm excited to see finally happen.