AstraZeneca and MedImmune, its global biologics research and development arm, today announced that the US Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for moxetumomab pasudotox, an investigational anti-CD22 recombinant immunotoxin and a potential new medicine for the treatment of adult patients with hairy cell leukaemia (HCL) who have received at least two prior lines of therapy. The FDA has granted the moxetumomab pasudotox BLA Priority Review status with a Prescription Drug User Fee Act date set for the third quarter of 2018.
The Phase III (‘1053’) moxetumomab pasudotox clinical trial met its primary endpoint of durable complete response in adult patients with relapsed or refractory HCL, for which there is currently no established standard of care and few treatments available. Results from the 1053 Phase III trial will be presented at a forthcoming medical meeting.
Priority Review is granted by the FDA to applications for medicines that, if approved, would offer a significant improvement in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions.
About Moxetumomab Pasudotox
Moxetumomab pasudotox (formerly CAT-8015 or HA22) is an investigational anti-CD22 recombinant immunotoxin and a potential new medicine with the opportunity to be a first-in-class treatment in the US for patients with relapsed or refractory HCL who have received at least two prior lines of therapy. Immunotoxins are a class of anticancer agents that combine the selectivity of antibodies to target drug delivery and the potency of toxins to kill target cancer cells. Moxetumomab pasudotox is composed of a binding portion of an anti-CD22 antibody fused to a toxin. CD22 is a B-lymphocyte restricted transmembrane protein with a higher receptor density in HCL cells relative to normal B cells, making it an attractive therapeutic target for the treatment of this cancer. After binding to CD22, the molecule is internalised, processed and releases its modified protein toxin that inhibits protein translation, leading to apoptotic cell death. Moxetumomab pasudotox has been granted Orphan Drug Designation by the FDA for the treatment of HCL.