Pharming Group Provides Update on Ongoing Regulatory Review of Leniolisib for the Treatment of APDS in the European Union
Leiden, the Netherlands, May 30, 2024 – Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) has recently shared 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 recent CHMP meeting held from May 27-30, Pharming received an updated List of Outstanding Issues (LoOI) from the CHMP. The LoOI confirmed the positive clinical benefit and safety of leniolisib, in line with the assessment by the Ad Hoc Expert Group (AEG), with one remaining chemistry, manufacturing, and controls (CMC) request. The CMC request pertains to the definition of regulatory starting materials used in the manufacturing process for leniolisib. Pharming has committed to meeting all of the CHMP’s specific requirements and has already initiated the necessary manufacturing activities requested by the CHMP.
Despite a delay to the European license, Sijmen de Vries, MD, Chief Executive Officer of Pharming, expressed optimism about the positive clinical benefit of leniolisib. The MAA for leniolisib was supported by findings from a multinational, triple-blind, placebo-controlled, randomized Phase II/III clinical trial, showing efficacy and safety in 31 patients diagnosed with APDS aged 12 years and older.
Leniolisib, currently available in the United States, is the first and only targeted treatment for APDS. The US Food and Drug Administration (FDA) approved leniolisib in March 2023 based on its clinical and manufacturing standards. As Pharming continues to work towards obtaining approval for leniolisib in Europe, they remain dedicated to addressing the unmet medical needs of individuals living with APDS.
In conclusion, Pharming Group N.V. remains committed to navigating the regulatory review process for leniolisib and providing a potential treatment option for individuals with APDS. The Company’s efforts to meet the CHMP’s requirements underscore their dedication to improving the lives of patients with rare diseases through innovative therapies.
Keywords: Pharming Group, leniolisib, APDS treatment, CHMP, European Medicines Agency, regulatory review
Reference:
https://www.pharming.com/