Lumoxiti™ (Moxetumomab pasudotox-tdfk) – 2018 FDA Approval

FDA Approval: 9/13/2018

Hairy cell leukemia (HCL) is a rare, slow-growing cancer of the blood in which the bone marrow produces too many abnormal B cells or lymphocytes. This compromises the bone marrow’s ability to produce healthy blood cells and platelets. Patients with HCL may experience enlargement of the spleen, increased susceptibility to life-threatening conditions such as infections and anemia, and general symptoms such as fever, fatigue, and weight loss. HCL comprises approximately 2% of all adults with leukemia, and approximately 1,000 individuals are diagnosed with HCL every year in the United States. While many patients will initially respond to treatment, up to 40% with HCL will relapse. After failing standard treatment, there was no standard of care and few compendia approved treatments available with efficacy for HCL, representing a significant unmet need for patients with multiple relapses.

Moxetumomab pasudotox-tdfk is an investigational anti-CD22 immunotoxin agent developed by AstraZeneca’s global biologics research and development arm, MedImmune, LLC for the treatment of relapsed or refractory hairy cell leukemia (HCL) in patients who have received at least two prior lines of therapy.  Moxetumomab pasudotox-tdfk was granted priority review by the FDA in April of 2018 for the treatment of adult patients with relapsed or refractory hairy cell leukemia who have received at least two prior lines of therapy. The drug candidate had an anticipated PDUFA date of September 3, 2018 and received approval on September 13, 2018.

For our complete review of Moxetumomab pasudotox-tdfk (Lumoxiti™):

Executive Summary of Evaluation:


Moxetumomab pasudotox

(AstraZeneca / MedImmune, LLC)







  Orphan Drug Designation


  Priority Review Designation


  FDA Approval


  Patent Expiration


Indications Relapsed/Refractory Hairy Cell Leukemia (HCL)

Approved (BLA)

Efficacy Relapsed/Refractory Hairy Cell Leukemia (HCL)

Efficacy reported in Phase III Trials



Clinical Trial data obtained at . Accessed: July 19, 2018.

“Epidemiology”. Hairy Cell Leukemia Foundation. . Accessed: July 19, 2018.

GlobalData, Pharma Intelligence Center. . Accessed: July 27, 2018.

Kreitman, RJ. Treating Patients with Relapsed/Refractory Hairy Cell Leukemia. The Hematologist. 2015 July; 12(4):4. Accessed: July 27, 2018.

“Moxetumomab pasudotox pivotal data in patients with previously-treated hairy cell leukaemia presented at the 2018 ASCO Annual Meeting”. AstraZeneca Media Websites. Published: June 4, 2018.  Accessed: July 25, 2018.

Troussard X, Cornet E. Hairy cell leukemia 2018: Update on diagnosis, risk‐stratification, and treatment. Am J Hematol. 2017 Dec; 92(12): 1382–1390. Accessed July 27, 2018.

U.S. Food & Drug Administration Drugs@FDA: FDA Approved Drug Products Product Labeling Accessed: September 21, 2018

Additional 2018 Pipeline Reviews are available in our pipeline library.

Please see the August 2018 FDA Actions Newsletter for additional information on recent FDA approvals

Coauthored by: Kristen Ciampi, PharmD

Kristen recently graduated with a PharmD degree from the University of Rhode Island in May of 2018, and is a Pharmacy Analytics Intern at RJ Health Systems.