Pharming Gets Priority Review for Leniolisib

The FDA has granted a Priority Review to Pharming’s New Drug Application (NDA) for leniolisib to treat patients with activated phosphoinositide 3-kinase delta syndrome (APDS) who are 12 years and older.

The rare immunodeficiency condition is caused by variants in either of two genes — PIK3CD or PIK3R1 — that regulate the maturation of white blood cells.

The investigational drug is an oral, selective phosphoinositide 3-kinase delta inhibitor the company gained through an August 2019 collaboration and licensing agreement with Novartis.

The NDA submission is supported by results from a phase 2/3 study, which met its endpoints of a reduction in lymph node size and correction of immunodeficiency in the target population.

The FDA has set a target decision date for the NDA of March 29, 2023.