US FDA approves Pharming’s immune disorder drug
2023.03.24 15:14
© Reuters. FILE PHOTO: FILE PHOTO: Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020. REUTERS/Andrew Kelly
(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.
