© Reuters. FILE PHOTO: Signage outside 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) approved a drug from Dutch firm Pharming Group on Friday 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 drug approved to treat activated phosphoinositide 3-kinase delta syndrome (APDS), a primary immunodeficiency that affects approximately 1 to 2 people in a million.
Joenja is expected to launch in the US in early April and will be available to ship in mid-April, Pharming said.
The approval comes a month after the European health regulator changed the 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 after the interim analysis.
APDS causes immune cells, B cells, and T cells to fail to function, making it difficult for people with the disorder to fight off bacterial and viral infections. It also exacerbates the risk of permanent lung damage and lymphoma over time.
