MARSEILLE, France—Innate Pharma SA announced today that the European Medicines Agency (EMA) has accepted the Marketing Authorization Application for Lumoxiti (moxetumomab pasudotox-tdfk), a first-in-class medicine indicated for 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 immunotoxin; 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 is not recommended in patients with severe renal impairment (CrCl ≤ 29 mL/min).
“If approved by the EMA, Lumoxiti will be the first treatment available in Europe for relapsed or refractory hairy cell leukemia patients in more than twenty years, potentially changing the standard of care for these patients. We are dedicated in addressing the unmet need in this rare form of cancer that can result in serious and life-threatening conditions, and as such, are hopeful we can bring this important medicine to patients in Europe as soon as possible,” said Pierre Dodion, M.D., executive vice president and chief medical officer of Innate Pharma.
The EMA filing is based on the final analysis of the pivotal Phase 3 trial of Lumoxiti, presented at ASH 2019. The trial enrolled 80 patients and was conducted across 34 sites in 14 countries.
These data showed that 36 percent (29/80) of the relapsed or refractory hairy cell leukemia patients achieved 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, pharmacokinetics and immunogenic potential.
The objective response rate (ORR) was at 75 percent. Eighty-one percent of patients with CR experienced eradication of minimal residual disease as reflected by MRD-negative status. In addition, there was a 61 percent probability that patients who achieved a CR would maintain it after five years.
The EMA filling acceptance follows the U.S. Food and Drug Administration (FDA) approval of Lumoxiti in September 2018. Lumoxiti has been granted Orphan Drug Designation by the FDA and the EMA for the treatment of r/r HCL. AstraZeneca currently holds the Biologics License Application holder for Lumoxiti in the U.S., and is also the marketing authorization applicant for the EU filing.