(Reuters) – The U.S. Food and Drug Administration (FDA) on Friday approved Dutch firm Pharming Group’s drug to treat a rare genetic disorder that leads to a weakened immune system, the company said.
Leniolisib, to be sold under the brand name Joenja, becomes the first approved drug to treat activated phosphoinositide 3-kinase delta syndrome (APDS), a primary immunodeficiency that affects about 1 to 2 people in a million.
Joenja is expected to launch in the U.S. in early April and will be available for shipment in mid-April, Pharming said.
The approval comes a month after the European health regulator shifted accelerated review of leniolisib to a standard one, citing the need for the company to submit additional data from an extension study that was conducted post-interim analysis.
APDS causes a lack of functioning immune cells, B cells and T cells, which makes it difficult for people with this disorder to fight off bacterial and viral infections. It also exacerbates the risk of permanent lung damage and lymphoma over time.
(Reporting by Mariam Sunny, Khushi Mandowara and Raghav Mahobe in Bengaluru; Editing by Shounak Dasgupta)
