Pharming Group provides updates on EMA regulatory review of leniolisib MAA and plans to file for UK regulatory approval

Leiden, The Netherlands, November 10, 2023: Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces that it has received a Day 180 Second List of Outstanding Issues (LoOI) from the European Medicines Agency’s (EMA) Committee for Human Medicinal Products (CHMP) regarding its Marketing Authorisation Application (MAA) for leniolisib for adult and pediatric patients 12 years of age and older with APDS, a rare primary immunodeficiency. Based on the timetable included in the CHMP Day 180 Second LoOI, and considering the CHMP’s decision to reschedule the Ad-Hoc Expert Group (AEG) meeting to the end of November, Pharming now expects the CHMP to issue its opinion on the leniolisib MAA in the first quarter of 2024.

Pharming now intends to file an MAA with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) through the International Recognition Procedure (IRP) which will replace the European Commission Decision Recognition Procedure (ECDRP) from January 1, 2024. Under the IRP, Pharming would file an MAA for leniolisib on the basis of the US FDA approval in the first quarter of 2024 and the MHRA would have 110 days from the date the IRP submission is validated to review and issue its decision.

About Activated Phosphoinositide 3-Kinase δ Syndrome (APDS)

APDS is a rare primary immunodeficiency that was first characterized in 2013. APDS is caused by variants in either one of two identified genes known as PIK3CD or PIK3R1, which are vital to the development and function of immune cells in the body. Variants of these genes lead to hyperactivity of the PI3Kδ (phosphoinositide 3-kinase delta) pathway, which causes immune cells to fail to mature and function properly, leading to immunodeficiency and dysregulation^1,2,3 APDS is characterized by a variety of symptoms, including severe, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.^4,5 Because these symptoms can be associated with a variety of conditions, including other primary immunodeficiencies, it has been reported that people with APDS are frequently misdiagnosed and suffer a median 7-year diagnostic delay.⁶ As APDS is a progressive disease, this delay may lead to an accumulation of damage over time, including permanent lung damage and lymphoma.^4-7 A definitive diagnosis can be made through genetic testing. APDS affects approximately 1 to 2 people per million worldwide.