Release Date: 29-Sep-2021
European Medicines Agency (EMA) has granted orphan designation for bomedemstat, a lysine-specific demethylase-1 (LSD1) inhibitor, for the treatment of essential thrombocythemia (ET).
It is given to investigational therapies that have the power to treat rare and life-threatening diseases that affect less than five in 10,000 patients in the European Union. It provides some benefits to companies, like clinical protocol advice, fee reductions, and ten years of protection from competition following regulatory approval.
As per the data of 26th European Hematology Association Virtual Congress, the bomedemstat was well-tolerated by patients with ET and showed promising clinical activity as a monotherapy in patients who have become resistant to or intolerant of one or more standard-of-care treatments. The Phase 2 clinical trial of bomedemstat for the treatment of ET continues to actively enroll globally.