The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to mavorixafor for the treatment of chronic neutropenia.
Neutropenia is a rare genetic, autoimmune condition characterized by low levels of granulocytes, specifically neutrophils. The condition is classified as chronic neutropenia when these low levels last more than three months. These neutrophils are retained in the bone marrow by the CXCR4/CXCL12 axis.
Mavorixafor is an oral CXCR4 antagonist that is designed to downregulate the CXCR4 receptor and mobilize functional neutrophils from the bone marrow into the blood. The investigational treatment is currently being evaluated in the global, pivotal phase 3 clinical trial (4WARD Study). This trial is specifically looking at the efficacy, safety, and tolerability of oral, once-daily mavorixafor in patients with congenital, acquired primary autoimmune, or idiopathic chronic neutropenia who are experiencing recurrent and/or serious infections.
The trial is a 52-week, randomized, double-blind, placebo-controlled, multicenter study hoping to enroll 150 participants. The primary endpoint is based on annualized infection rate and positive absolute neutrophil count (ANC) response.
Current treatment for chronic neutropenia is injectable human recombinant granulocyte-colony stimulating factor (G-CSF). However, this therapy is associated with many side effects such as bone pain, splenomegaly, thrombocytopenia, glomerulonephritis, vasculitis, osteoporosis, and with an increased risk of leukemia at long-term high doses. Due to these limitations, there are many unmet needs in this patient population.
The Fast Track designation will facilitate and streamline the development and expedite the review of mavorixafor for those with chronic neutropenia.
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To learn more about chronic neutropenia and other rare hematologic disorders, visit https://checkrare.com/diseases/hematologic-disorders/