The European Medicines Agency (EMA) grants orphan drug designation status to medicines that are intended to treat rare diseases. The designation makes the approval process simpler and less costly for such medicines.
Pracinostat, which was developed by the Helsinn Group and MEI Pharma, is intended to treat acute myeloid leukaemia (AML). The investigational drug candidate is currently in a Phase 3 study for the treatment of AML in adult patients who cannot undergo chemotherapy. The orphan drug designation was based on positive Phase 2 study results, in which 50 AML patients were treated with pracinostat.
Helsinn CEO Riccardo Braglia welcomed the orphan drug designation status: “This decision encourages us to continuously dedicate significant resources to accelerate our clinical trial programme, with a goal of helping patients who are fighting rare and difficult-to-treat diseases.”
Headquartered in Lugano, the Helsinn Group specializes in the development of products for the treatment of different forms of cancer. MEI Pharma from San Diego, CA, focuses on the clinical development of novel therapies for cancer.