Lumoxiti (moxetumomab pasudotox-tdfk) is a marketed product, in-licensed from AstraZeneca’s for the treatment of hairy cell leukemia (“HCL”). Read the press release.
Lumoxiti is a CD22-directed immunotoxin and a first-in-class treatment in the US for adult patients with relapsed or refractory (r/r) HCL who have received at least two prior systemic therapies, including treatment with a purine nucleoside analog. Lumoxiti is not recommended in patients with severe renal impairment (CrCl ≤ 29 mL/min). It comprises the CD22 binding portion of an antibody fused to a truncated pseudomonas exotoxin. The toxin inhibits protein synthesis and ultimately triggers apoptotic cell death.
Lumoxiti received U.S. FDA approval in September 2018 and has been granted Orphan Drug Designation by the FDA and the European Medicines Agency (EMA) for the treatment of r/r HCL. The Market Authorization Application for Lumoxiti was accepted for review by the EMA.
More information about Lumoxiti are available at https://www.lumoxiti.com/
Hairy cell leukemia (HCL) is a rare, chronic, and slow-growing leukemia in which the bone marrow overproduces abnormal B cell lymphocytes. HCL can result in serious conditions, including infections, bleeding and anemia. Approximately 1,000 people are diagnosed with HCL in the US each year. HCL accounts for up to 3% of all adult leukemias. While many patients initially respond to treatment, 30% to 40% will relapse five to ten years after their first treatment. With no established standard of care and very few treatments available, there remains significant unmet medical need for people with relapsed or refractory HCL.
The approval of Lumoxiti was based on data from the AstraZeneca-sponsored, open-label ‘1053’ trial, which was a single-arm, multicenter Phase III clinical trial assessing the efficacy, safety, immunogenicity and pharmacokinetics of Lumoxiti monotherapy in patients with relapsed or refractory HCL who have received at least two prior therapies, including one purine nucleoside analog. The trial enrolled 80 patients and was conducted across 34 sites in 14 countries. The primary endpoint was durable complete response (CR), defined as CR with hematologic remission (blood count normalization) for more than 180 days. Secondary endpoints included overall response rate, relapse-free survival, progression-free survival, time to response, safety, pharmacokinetic and immunogenic potential.
Patients may gain access to unapproved or investigational medicines through clinical trials or early access programs (EAPs) before they are approved by regulatory authorities. EAPs allow a limited group of patients, who meet specific criteria, to have access to a specific investigational medicine. EAPs may have a variable designation in the different countries; for example, they are called authorization for temporary use (ATU) in France.
An EAP for Lumoxiti is available in France, Germany, UK, Spain, Italy and Switzerland. Lumoxiti is accessible through an ATU in France. For further information about this program in your area, please contact our Medical Affairs team at email@example.com, or by phone at +33(0)977 40 40 41.