The first new drug in 20 years! AstraZeneca immunotoxin lumoxiti applied for listing in the European Union to treat hair cell leukemia (HCl)

January 4, 2020 / BIOON / -- the French pharmaceutical company, innate Pharma SA, recently announced that the European Drug Administration (EMA) has accepted lumoxiti (moxetummab Pasudotox's marketing authorization application (MAA) is the first anti-cd-22 recombinant immunotoxin for the treatment of adult patients with relapsed or refractory hair cell leukemia (HCl) who have previously received at least two kinds of systemic therapy (including purine nucleoside analogues) treatment failure In the United States, lumoxiti was approved for these indications in September 2018 This approval makes lumoxiti the first drug approved to treat HCl in the past 20 years, marking a major milestone in the clinical treatment of HCl Lumoxiti is a product of AstraZeneca In October 2018, innate pharma and AstraZeneca reached an extended cooperation agreement to promote the development of new oncology pipeline drugs According to the terms of the cooperation agreement, innate Pharma will act for lumoxiti AstraZeneca is the holder of lumoxiti's biological products licensing application (BLA) in the U.S market and the marketing authorization applicant filed in the European Union Lumoxiti's approval in the US and MAA submission in the EU are based on data from the critical phase III clinical study (study 1053) This single arm, multicenter study was conducted in 80 adult patients with recurrent or refractory HCl who had previously received at least two regimens to evaluate the efficacy and safety of lumoxiti single drug treatment The study was conducted in 34 treatment centers in 14 countries The results of this study have been published at the annual meeting of the American Society of Hematology (ash) in 2019 The data show that the overall response rate (ORR) of single drug treatment of lumoxiti is 75%, and the long-term complete response rate (CR) is 36% (29 / 80) It is defined as the complete response (CR) of hematological response maintained for at least 180 days 81% of Cr patients experienced the eradication of minimal residual lesions, i.e MRD negative state In addition, patients with Cr were 61% likely to maintain CR after 5 years HCl is a rare, incurable, slow-moving lymphoproliferative chronic leukemia, characterized by anemia, hemorrhage, splenomegaly, peripheral blood and bone marrow with a large number of white blood cells with irregular margin and pseudopodiform or fibroid protrusion HCl can cause serious, life-threatening consequences, including severe infection, bleeding, and anemia Immunotoxin is a kind of anticancer agent, which uses the selective antibody to target drug delivery and the ability of toxin to kill cancer cells Lumoxiti is composed of a binding domain of anti CD22 antibody and toxin CD22 is a type I transmembrane protein mainly expressed in mature B lymphocytes and plays an important role in B cell signaling Compared with normal B cells, there is a higher density of CD22 on HCl cells, which makes it a very attractive target for treatment of HCl When lumoxiti is combined with CD22, it will be internalized, processed and released by cells, which will inhibit the translation of proteins in cells and lead to apoptosis In the United States and the European Union, lumoxiti has been granted orphan drug status (odd) for the treatment of HCl, and has also been granted fast track qualification in the United States At present, there is no mature standard nursing plan for HCl Although many patients show remission at the beginning of treatment, up to 30% - 40% of patients will relapse within 5-10 years after the first treatment However, in the subsequent treatment, the time of remission is shortened and toxicity is accumulated, so there are few treatment options Lumoxiti represents a very promising non chemotherapy drug, which is expected to solve the significant unmet medical needs of patients with recurrent or refractory HCl In terms of drug use, the recommended dose of lumoxiti is 0.04mg/kg body weight, the time of intravenous infusion is more than 30 minutes, and the drug is given on the first, third and fifth day of a cycle at 28 days, until 6 cycles of treatment or disease progression or unacceptable toxicity It should be noted that lumoxiti is not recommended for patients with severe renal impairment (creatinine clearance [CrCl] < 29ml) The European Medicines Agency accepts the regulatory submission for lumoxiti in relapsed or refractory hairy cell leukemia