FDA grants Fast Track designation to givinostat for polycythemia vera

On May 6, 2025, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to givinostat, an orally administered histone deacetylase inhibitor for the treatment of patients with polycythemia vera (PV).^1,2 

Givinostat previously received orphan drug designation from the FDA and the European Medicines Agency (EMA) for PV, and marketing authorizations from the FDA and the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) for Duchenne muscular dystrophy. The EMA’s Committee for Medicinal Products for Human Use (CHMP) also gave a positive opinion for givinostat in Duchenne muscular dystrophy in April 2025, with a decision expected from the European Commission in July 2025.^1,2 

The randomized, open-label phase III GIV-IN PV trial (NCT06093672), which will evaluate the efficacy and safety of givinostat compared with hydroxyurea in patients with JAK2V617F-positive high-risk PV, is currently enrolling.¹ The primary endpoint for the trial is the proportion of patients achieving a response at Week 48 based on hematocrit level, white blood cell and platelet count levels, and spleen size. Secondary endpoints include the proportion of patients achieving a complete hematologic response (CHR) at Week 48, time from randomization to first CHR, proportion of patients with a normal spleen size at Week 48, and safety and tolerability up to Week 48. Estimated study completion will be in July 2026.