Italfarmaco's Givinostat Gains FDA Fast Track Status for Polycythemia Vera

Italfarmaco S.p.A., based in Milan, Italy, has announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to their drug, givinostat, for treating polycythemia vera (PV). This is a rare type of blood cancer with limited treatment options. The decision by the FDA to expedite the development of givinostat highlights the urgent need for new and effective treatments for PV and recognizes the potential impact that givinostat could have on patient care, according to Paolo Bettica, MD, PhD, Chief Medical Officer at Italfarmaco Group. He expressed the company's anticipation in collaborating closely with the FDA as they advance towards the completion of their ongoing Phase III clinical trial.

Givinostat is a histone deacetylase inhibitor (HDACi) administered orally. It has potential applications in treating both neuromuscular disorders and cancers. The function of HDACis involves modulating essential cellular pathways that control gene expression, which can provide therapeutic benefits across various diseases. The focus is currently on givinostat’s potential to manage PV, a rare blood disorder where there is an overproduction of erythroid, myeloid, and megakaryocytic components in the bone marrow. PV typically results in symptoms like headaches, weakness, and itching, and can lead to severe complications such as strokes, heart attacks, and deep vein thrombosis, which are major causes of death. Additionally, PV may progress to myelofibrosis or acute myeloid leukemia in some cases.

The mechanism of givinostat aims to address abnormal gene expression, potentially assisting in controlling excessive cell proliferation, particularly those driven by mutations like JAK2V617F, commonly seen in PV patients. This approach is intended to reduce the disease burden, alleviate symptoms, and enhance long-term outcomes for patients. The Phase III clinical trial (NCT06093672) for givinostat is actively enrolling participants, with clinical sites established in locations across Europe, the UK, Israel, and North America, and more sites anticipated to open soon.

The FDA's Fast Track designation aims to facilitate the development and quicken the review process of new drugs or biologics that target serious or life-threatening conditions while addressing unmet medical needs. This initiative seeks to streamline the drug development and review process by promoting early and frequent interactions between the FDA and pharmaceutical companies.

Furthermore, givinostat has been granted orphan drug designation by both the FDA and the European Medicines Agency (EMA) for PV. It also has marketing authorizations under the brand name Duvyzat® for treating Duchenne muscular dystrophy, granted by the FDA and the UK's Medicines and Healthcare products Regulatory Agency. A positive opinion has been adopted by the European Medicines Agency Committee for Medicinal Products for Human Use, with other regulatory processes still underway.

Italfarmaco, established in 1938 in Milan, operates as a global pharmaceutical leader, having developed and successfully brought numerous pharmaceutical products to market worldwide. With a presence in over 60 countries, Italfarmaco excels in research, product development, production, and commercialization in various therapeutic fields, including immuno-oncology, gynecology, neurology, cardiovascular disease, and rare diseases. Their rare disease unit is engaged in programs targeting conditions such as Duchenne muscular dystrophy, Becker muscular dystrophy, amyotrophic lateral sclerosis, and polycythemia vera.