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.
About Pharming Group N.V.
Pharming Group N.V. is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. Pharming is commercializing and developing an innovative portfolio of protein replacement therapies and precision medicines, including small molecules, biologics, and gene therapies that are in early to late-stage development. Pharming is headquartered in Leiden, Netherlands, and has employees around the globe who serve patients in over 30 markets in North America, Europe, the Middle East, Africa, and Asia-Pacific.
September 29, 2022
