The FDA ( U.S. Food and Drug Administration ) has approved Lumoxiti ( Moxetumomab pasudotox-tdfk ) injection for intravenous use for the treatment of adult patients with relapsed or refractory hairy cell leukemia ( HCL ) who have received at least two prior systemic therapies, including treatment with a purine nucleoside analog.
Lumoxiti is a CD22-directed cytotoxin and is the first of this type of treatment for patients with hairy cell leukemia.
Hairy cell leukemia is a rare, slow-growing cancer of the blood in which the bone marrow makes too many B cells ( lymphocytes ), a type of white blood cell that fights infection.
Hairy cell leukemia is named after these extra B cells which look hairy when viewed under a microscope.
As the number of leukemia cells increases, fewer healthy white blood cells, red blood cells and platelets are produced.
The efficacy of Lumoxiti was studied in a single-arm, open-label clinical trial of 80 patients who had received prior treatment for hairy cell leukemia with at least two systemic therapies, including a purine nucleoside analog.
The trial measured durable complete response ( CR ), defined as maintenance of hematologic remission for more than 180 days after achievement of complete response.
Thirty percent of patients in the trial achieved durable complete response, and the overall response rate ( number of patients with partial or complete response to therapy ) was 75%.
Common side effects of Lumoxiti include infusion-related reactions, swelling caused by excess fluid in body tissue ( edema ), nausea, fatigue, headache, fever ( pyrexia ), constipation, anemia and diarrhea.
The prescribing information for Lumoxiti includes a Boxed Warning to advise health care professionals and patients about the risk of developing capillary leak syndrome, a condition in which fluid and proteins leak out of tiny blood vessels into surrounding tissues.
Symptoms of capillary leak syndrome include difficulty breathing, weight gain, hypotension, or swelling of arms, legs and/or face.
The Boxed Warning also notes the risk of hemolytic uremic syndrome, a condition caused by the abnormal destruction of red blood cells.
Patients should be made aware of the importance of maintaining adequate fluid intake, and blood chemistry values should be monitored frequently.
Other serious warnings include: decreased renal function, infusion-related reactions and electrolyte abnormalities.
Women who are breastfeeding should not be given Lumoxiti.
The FDA granted this application Fast Track and Priority Review designations.
Lumoxiti also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. ( Xagena2018 )
Source: FDA, 2018