A leukaemia drug that opened a whole new class just won the prize they call biotech's Nobel for rare diseases

Revuforj (revumenib) works differently from every leukaemia drug before it: it blocks a protein interaction — the menin pathway — that certain aggressive leukaemias depend on. This week the Galien Foundation named it the 2026 Prix Galien USA Best Product for Rare/Orphan Diseases. It is the first and only menin inhibitor approved for adults and children from one year old with relapsed or refractory acute leukaemia carrying either of two genetic changes, KMT2A translocations or NPM1 mutations — patients whose cancers had returned and whose options were nearly gone.

SDG 3 · Good health and well-being — Mechanism: tech innovation