March 24 Quick Takes: FDA approves Pharming’s immunodeficiency therapy

FDA approved PI3Kδ inhibitor Joenja leniolisib from Pharming Group N.V. (Euronext:PHARM) to treat activated PI3Kδ syndrome (APDS), an ultra-rare primary immunodeficiency. Pharming licensed the therapy from Novartis AG (SIX:NOVN; NYSE:NVS) in 2019 for $20 million up front; the pharma and a third party are due $10.5 million for approval and first commercial sale, and Novartis eligible for $190 million in milestones, plus royalties. Pharming said Novartis may purchase a priority review voucher tied to the approval for “a small minority share” of the voucher’s value.

Incyte Corp. (NASDAQ:INCY) received a complete response letter from FDA for a once-daily tablet formulation of ruxolitinib, which the company markets in a twice-daily formulation as Jakafi to treat myelofibrosis, polycythemia vera and graft-versus-host disease. The company said that although FDA acknowledged that the daily JAK1/JAK2 inhibitor showed bioequivalence in a pivotal study, there were undisclosed “additional requirements” precluding its approval...