Pharming provides update on EMA regulatory review of leniolisib for APDS in Europe
EMA Marketing Authorisation Application for leniolisib changed to standard review timetable
Leiden, The Netherlands, February 16, 2023
Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces that the European Medicines Agency's (EMA) Committee for Human Medicinal Products (CHMP) has decided to shift its assessment of the Marketing Authorisation Application (MAA) for leniolisib to a standard review timetable. Leniolisib is an oral selective phosphoinositide 3-kinase delta (PI3Kd) inhibitor under regulatory review as a treatment for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, in adolescents and adults 12 years of age and older.
Pharming has received the list of questions from EMA which included a request to submit updated data from the ongoing long-term extension study collected after the interim analysis included in the original MAA. Based on the timetable for our responses, Pharming expects that CHMP will issue its opinion on the leniolisib MAA in 2H 2023.
Sijmen de Vries, CEO of Pharming commented:
"We are continuing to work with EMA through the MAA review process and remain dedicated to seeking regulatory approval of leniolisib within the European Economic Area. In the US, the FDA's priority review of the leniolisib NDA remains on track, with a PDUFA goal date of March 29, 2023. We remain focused on achieving regulatory approvals and bringing leniolisib to patients living with APDS worldwide."