On 13 September 2018, the US Food and Drug Administration(FDA) announcedthe approval of moxetumomab pasudotox-tdfk (Lumoxiti) intravenous injection for the treatment of adult patients with relapsed or refractory (R/R) hairy cell leukemia (HCL) after two prior lines of systemic therapies, including treatment with a purine nucleoside analog.
Moxetumomab pasudotox-tdfk is a CD22-directed cytotoxin that was granted priority reviewby the FDA on 03 April 2018. The approval follows data from the phase III trial (NCT01829711)in which the overall response rate was 75% (95% CI: 64,84) and durable complete response rate of 30% (95% CI: 20,41).
The FDA noted that common side effects of Moxetumomab pasudotox-tdfk included infusion-related reactions, swelling, nausea, fatigue, headache, fever, constipation, anemia and diarrhea. There is a risk of developing capillary leak syndrome and haemolytic uremic syndrome. Additionally, moxetumomab pasudotox-tdfk is not recommended in patients with severe renal impairment.