Givinostat Wins U.S. FDA Fast Track Designation for Patients With PV

Treatment with givinostat has received fast track designation from the United States Food and Drug Administration (FDA) for patients with polycythemia vera (PV), a rare blood cancer, for which treatment options are limited, according to a news release from Italfarmaco S.p.A.

Fast track designation, according to the news release, is a process intended to support the development and quicken the review of new drugs or biologics which aim at treating serious or life-threatening conditions — such as cancer — with unmet medical needs. This designation seeks to streamline the development and review process done by the FDA by promoting early and ongoing communication between the FDA and pharmaceutical companies.

“The FDA decision to grant givinostat fast track designation underscores the urgent need for innovative treatments for PV and highlights the potential of givinostat to make a meaningful difference,” Dr. Paolo Bettica, chief medical officer at Italfarmaco Group, said in the news release. “We look forward to working closely with the FDA as we plan for completion of our phase 3 clinical trial.”

The agent under investigation is used through oral administration and is a histone deacetylase inhibitor; it has potential use for patients with neuromuscular disorders and in oncology treatment. Additionally, histone deacetylase inhibitors work by regulating how certain genes are turned on or off in cells, offering potential therapeutic benefits across a range of diseases. With this goal in mind, givinostat is currently being investigated in patients with PV.

PV is a rare blood cancer which causes the overproduction of too many red and white blood cells, as well as platelets (erythroid, myeloid, and megakaryocytic components) in the bone marrow. The presence of PV can lead to symptoms like headache, weakness and itching. In severe cases, PV can cause things like stroke, heart attack or blood clots (deep vein thrombosis), which can be life-threatening. Patients with PV are also at risk of the disease progressing to myelofibrosis or acute myeloid leukemia.

Givinostat works by targeting and modulating abnormal gene activity that causes certain cells to grow too quickly; this is driven by a genetic mutation called JAK2V617F which is found in patients with PV. By targeting this mutation, givinostat may slow down the production of excess blood cells and reduce disease burden. This approach is intended to lessen the effects of the disease, relieve symptoms and support better long-term health.

In Europe, the UK, Israel and North America, a phase 3 trial is currently enrolling patients with PV to be treated with givinostat. Importantly, more clinical sites open are expected to open expected soon, the news release relays.

Notably, givinostat was granted orphan drug designation by the FDA and the European Medicines Agency for patients with PV. Additionally, the agent, when marketed as Duvyzat, has received marketing authorizations for Duchenne muscular dystrophy from both the FDA and the U.K. Medicines and Healthcare products Regulatory Agency. The agent also received a positive opinion issued by the Committee for Medicinal Products for Human Use and additional regulatory reviews currently in progress.

Orphan drug designation from the FDA is a special status which is granted to drugs which aim to treat, prevent or diagnose a rare diseases or condition, such as PV, according to the United States FDA official website, fda.gov. Notably, this status is intended to provide drug developers with incentives to encourage research and development of treatments.

For more information regarding the phase 3 trial, please visit www.clinicaltrials.gov using the identifier: NCT06093672.

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