- On September 13, the FDA approved moxetumomab pasudotox-tdfk for the treatment of adult patients with relapsed/refractory (R/R) hairy cell leukemia (HCL) who have received at least 2 prior systemic therapies, including treatment with a purine nucleoside analog.
Why this matters
- An unmet need has persisted for a new therapeutic option offering a greater rate and durability of response and reduced toxicity vs purine nucleoside analogs, and with the ability to eradicate minimal residual disease (MRD), unlike current targeted agents.
- The approval was based on results of a clinical trial of 80 patients with R/R HCL.
- Primary endpoint: durable complete response (CR), defined as proportion of patients maintaining hematologic remission for >180 days after CR.
- Funding: MedImmune; AstraZeneca Pharmaceuticals.
- 30% (95% CI, 20.3%-41.3%) durable CR.
- 75% (95% CI, 64.1%-84.0%) overall response rate.
- 80% achieved hematological remission in approximately 1 month.
- 41% (95% CI, 30.4%-52.8%) achieved CR.
- Median duration of CR was not reached in patients with MRD negativity.
- The most common adverse effects included infusion-related reactions, edema, nausea, and fatigue.
- Single-arm, open-label study.
Prescribing Information, click here.