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.

Sijmen de Vries, MD, Chief Executive Officer of Pharming, commented:

“While we are understandably disappointed by the delay to the European license, we are pleased that the CHMP determined the clinical benefit of leniolisib to be positive. The efficacy and safety demonstrated in clinical trials and ongoing real-world experience, with over 300 patient-years of treatment, support that leniolisib fills an unmet medical need. We will continue to work closely with the EMA and CHMP to obtain approval for leniolisib in Europe for people living with APDS. In the meantime, all our clinical development and early access programs will continue.”