Pharming Group N.V. announced that it has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kd) inhibitor, as a treatment for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, in adults and adolescents 12 years or older. On August 1, 2022, Pharming announced the leniolisib MAA was granted accelerated assessment by EMA's Committee for Medicinal Products for Human Use (CHMP). The accelerated assessment reduces the review timeframe from 210 days to 150 days.

Upon request, the EMA will grant an accelerated assessment of an MAA if they decide the product is of major interest for public health, and in particular, from the viewpoint of therapeutic innovation. Marketing authorisation for leniolisib in the EEA is anticipated in H1 2023. The MAA is supported by positive data from a Phase II/III study of leniolisib, announced on February 2, 2022, which met its co-primary endpoints of reduction in lymph node size and correction of immunodeficiency in the target population.

Furthermore, safety data from the study showed that leniolisib was well tolerated by participants. Also submitted as part of the MAA were data from a long-term, open-label extension clinical trial in patients with APDS treated with leniolisib.