The U.S. Food and Drug Administration (FDA) recently granted Breakthrough Therapy Designation (BTD) to IPN60340, developed by the French pharmaceutical company Ipsen. This drug is intended for use in combination with venetoclax and azacitidine (Ven-Aza) for the first-line treatment of elderly patients with acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy. Acute myeloid leukemia is an aggressive blood cancer that particularly affects the elderly population.

IPN60340 is a first-in-class monoclonal antibody uniquely designed to target the BTN3A molecule. BTN3A is a key immune regulatory molecule widely expressed in the field of cancer. By precisely targeting this molecule, IPN60340 offers the potential to provide a new treatment option for patients with acute myeloid leukemia. Following this announcement, Ipsen's stock price rose by 5.6% on the day, reaching €134.6, reflecting the market's positive response to the drug's development progress.

The Breakthrough Therapy Designation is a special mechanism established by the FDA to expedite the development and review of drugs intended to treat serious or life-threatening conditions and for which preliminary clinical evidence indicates substantial improvement over existing therapies. In addition to receiving this FDA designation, IPN60340 also received Orphan Drug Designation from both the U.S. Food and Drug Administration and the European Medicines Agency in July 2025, further highlighting its potential in the field of rare disease treatment.