EC Grants Orphan Drug Designation to Pharming’s Leniolisib

The European Commission (EC) has granted orphan drug designation to Pharming Group for its treatment of activated phosphoinositide 3-kinase delta syndrome (APDS), leniolisib, based on the positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicine Agency (EMA).

“We are pleased to have received orphan drug designation from the EC, an important milestone in the development of leniolisib for the treatment for APDS, an ultra-rare and debilitating disease,” said Sijmen de Vries, CEO of Pharming, in an Oct. 21, 2020 press release. “With no currently approved treatment, leniolisib has the potential to address a significant unmet need for patients with APDS. Leniolisib is currently being studied in a registration-enabling Phase II/III trial and remains, subject to regulatory approval, on track to launch in H2 2022.”

Leniolisib is a small molecule phosphoinositide 3-kinase delta inhibitor with immunomodulating and potentially anti-neoplastic activities. It has been granted orphan drug designation by the US Food and Drug Administration in January 2018 for the treatment of APDS or p110δ-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency.