On 13 September 2018, the US Food and Drug Administration (FDA) announced the 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 review by 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.