FDA approves new treatment for hairy cell leukemia


  • David Reilly
  • Oncology drug update
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Takeaway

  • 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. 

Study design

  • 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. 

Key results

  • 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.

Limitations

  • Single-arm, open-label study.

Prescribing Information, click  here.

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