Helsinn obtains orphan designation for pracinostat to treat AML

Swiss pharmaceutical group Helsinn and US-based oncology firm MEI Pharma have obtained orphan drug designation from the European Medicines Agency (EMA) for investigational drug candidate pracinostat for the treatment of acute myeloid leukaemia (AML).

Pracinostat is an oral histone deacetylase (HDAC) inhibitor that is currently under Phase III clinical trial in combination with azacitidine for adult AML patients who are eligible for induction chemotherapy.

The drug candidate received the US Food and Drug Administration (FDA) breakthrough therapy designation for use with azacitidine in newly diagnosed AML patients aged 75 years and above or not suitable for intensive chemotherapy.

Helsinn Group vice-chairman and CEO Riccardo Braglia said: "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, such as AML and, at present, have very few treatment options."

"This represents another important milestone in the global development strategy for Pracinostat."

The EMA’s decision is based on the scarcity of treatments for this disease, and positive Phase II data from 50 newly diagnosed patients aged 65 and older, and unfit for induction chemotherapy.

According to the results, the combination of pracinostat and azacitidine demonstrated median overall survival of 19.1 months and 62% one-year survival.

MEI Pharma president and CEO Daniel Gold said: "This designation from the EMA recognises the potential that Pracinostat holds in addressing a significant unmet need for those suffering with AML.

"This represents another important milestone in the global development strategy for Pracinostat."

The firm entered an exclusive licence, development and commercialisation agreement with Helsinn in August 2016 for pracinostat in AML and potential additional indications.