Pharming Leniolisib Launch Will Require Identifying Patients With Ultra Rare APDS

Pharming Group N.V. is on the cusp of scoring its second product approval, with the US Food and Drug Administration action date for leniolisib in activated phosophoinositide 3-kinase delta (PI3K-delta) syndrome (APDS) on the horizon. The company sees the rare disease drug as potentially providing an opportunity to add a new revenue stream and expand its commercial footprint globally, and to that end has been building up its commercial infrastructure ahead of the launch.

Scalable operations in both the US and Europe will position the company to bring in additional rare disease assets, CEO...