Financial Performance - Total revenues for Q3 2025 increased by 30% to US$97.3 million[13, 43] - RUCONEST® revenues in Q3 2025 reached US$82.2 million[13, 22] - Joenja® revenues in Q3 2025 amounted to US$15.1 million[13, 27] - Operating profit significantly grew to US$16.0 million in Q3 2025[13, 43] - Cash flow from operations was US$32 million in Q3 2025[14, 41] - Total revenues for 9M 2025 grew 33% to US$269.6 million[44, 45] Business Updates and Outlook - 2025 revenue guidance raised to US$365-375 million due to strong RUCONEST® performance[14, 51] - RUCONEST® U S volume growth was +24% in 3Q25 and +28% in 9M25[22] - There are 116 US patients on Joenja® representing a +25% increase vs 3Q24[27] - FDA filing for Leniolisib for 4-11 year old APDS patients is based on Phase III data and has a PDUFA date of Jan 31, 2026[28]

Core Viewpoint - Pharming Group N.V. announced that the FDA has accepted its supplemental New Drug Application for leniolisib, aimed at treating children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS), with a target action date of January 31, 2026 [1][7]. Group 1: FDA Approval Process - The supplemental New Drug Application is based on positive results from a Phase III study showing improvements in lymphadenopathy and naïve B cells over 12 weeks [2]. - The FDA has granted Priority Review status to the application, indicating that the treatment could offer significant improvements in effectiveness or safety for serious conditions [3]. Group 2: Disease Background - APDS is a rare primary immunodeficiency characterized by immune dysregulation and recurrent infections, often leading to severe complications such as lung damage and lymphoma [5]. - The condition affects approximately 1 to 2 individuals per million globally, and patients often experience a median diagnostic delay of 7 years [5]. Group 3: Treatment Details - Leniolisib is an oral PI3Kδ inhibitor, previously approved for patients aged 12 and older, and is the first targeted treatment for APDS [6][4]. - The drug has shown statistically significant improvements in clinical trials, and its safety and tolerability have been supported by long-term data [6]. Group 4: Future Developments - Leniolisib is currently under regulatory review in multiple regions, including the European Economic Area, Japan, and Canada, for its use in APDS [6]. - The company is also evaluating leniolisib in additional clinical trials for other primary immunodeficiencies with immune dysregulation [8].

Financial Performance - Total revenues for Q2 2025 increased by 26% compared to Q2 2024 [16] - RUCONEST® revenues in Q2 2025 reached $80.4 million, up from $63.0 million in Q2 2024 [14, 23] - Joenja® revenues in Q2 2025 were $12.8 million, compared to $11.1 million in Q2 2024 [14, 28] - Adjusted operating profit for Q2 2025 was $12.9 million, compared to a loss of $3.1 million in Q2 2024 [14] - The company is raising its 2025 revenue guidance to $335-350 million [16] - Revenues for 1H 2025 increased by 33% to $172.315 million, compared to $129.679 million in 1H 2024 [55, 63] Commercial Updates - RUCONEST® U S volume growth increased by 27% in Q2 2025 and 31% in 1H 2025 [23] - The number of U S patients on Joenja® increased by 25% compared to Q2 2024, reaching 114 patients in Q2 2025 [28] Research and Development - A study uncovered >100 new variants leading to PI3Kδ hyperactivity (GOF variants) [38] - Data suggests that VUS patients with these GOF variants should be reclassified as APDS [39] - APDS may be up to 100x more prevalent than previously estimated [40]