The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to givinostat, an oral histone deacetylase inhibitor (HDACi) developed by Italfarmaco S.p.A., for the treatment of polycythemia vera (PV), a rare hematologic malignancy. This designation aims to expedite the development and review of drugs that address serious conditions with unmet medical needs.
PV is characterized by the overproduction of red blood cells, often due to the JAK2V617F mutation, leading to symptoms such as headaches, weakness, and itching, and increasing the risk of severe complications like stroke and heart attack. Givinostat targets and modulates abnormal gene expression, potentially controlling excessive cell proliferation associated with PV.
The ongoing Phase III clinical trial (NCT06093672) is evaluating the efficacy and safety of givinostat compared to hydroxyurea in high-risk PV patients. Clinical sites are active in Europe, the UK, Israel, and North America, with additional sites expected to open soon.
Givinostat has previously received orphan drug designation from both the FDA and the European Medicines Agency (EMA) for PV. It is also approved under the brand name Duvyzat® for the treatment of Duchenne muscular dystrophy.
For more information, please refer to the official press release: Italfarmaco Announces U.S. FDA Grants Fast Track Designation to Givinostat in Treatment of Polycythemia Vera.
