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Nonprofit Buys Experimental Leukemia Drug to Keep Pediatric Access Open

Preserving limited doses of luveltamab tazevibulin for children with a rare, deadly AML subtype, Blood Cancer United aims to keep treatment available without commercializing it.

Overview

  • Blood Cancer United announced on June 11 that it bought the remaining supply of luveltamab tazevibulin and the investigational new drug (IND) rights so it can run a compassionate‑use program and distribute the medicine at no cost while supplies last.
  • Luvelta is an antibody‑drug conjugate that targets the folate receptor FOLR1 and was first developed for adult cancers but showed early, meaningful remissions in some children with CBFA2T3‑GLIS2–rearranged acute myeloid leukemia.
  • The supply is finite and currently expires in 2028, so the group plans stability testing to seek a longer shelf life and says it will evaluate but not commit to commercial development.
  • Individual cases, including a child treated under the earlier compassionate‑use program who remains in remission, illustrate the drug’s potential to bridge patients to potentially curative bone marrow transplants.
  • Advocates say the deal highlights gaps in incentives and regulatory pathways for rare pediatric drugs and could serve as a model for nonprofit stewardship, with the next steps hinging on trial work, FDA guidance, and efforts to secure longer‑term production or alternative therapies.