Pharming Group N.V. announced an update on the ongoing review of its Marketing Authorisation Application (MAA) for leniolisib for the treatment of adult and pediatric patients 12 years of age and older with activated phosphoinositide 3-kinase delta syndrome (APDS) by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). Following the May 27-30 CHMP meeting, Pharming received an updated List of Outstanding issues (LoOI) from the CHMP. The LoOI affirmed the positive clinical benefit and safety of leniolisib, in agreement with the assessment by the Ad Hoc Expert Group (AEG), and included one remaining chemistry, manufacturing and controls (CMC) request.

The CMC request relates to the definition of regulatory starting materials used in the manufacturing process for leniolisib. As Pharming is committed to meeting all of the CHMP?s specific requirements, additional data and quality controls were provided and Pharming proposed implementation of the CMC request post-approval. The CHMP requested that this work be completed pre-approval and has granted Pharming an extension to January 2026 to submit a response.

Pharming has already initiated the manufacturing activities requested by the CHMP, which it plans to complete prior to this deadline. The MAA for leniolisib was based on findings from a multinational, triple-blind, placebo-controlled, randomized Phase II/III clinical trial, which met both its co-primary endpoints. The trial evaluated efficacy and safety in 31 patients diagnosed with APDS aged 12 years and older.

Also submitted as part of the application were data from a long-term, open-label extension clinical trial in which 37 patients received leniolisib for a median of three years. Leniolisib is currently available commercially in the United States. The US Food and Drug Administration (FDA) approved leniolisib in March 2023, based on its assessment that leniolisib met clinical and manufacturing standards.