US FDA approves Pharming's immune disorder drug (updated)
24/03/2023 18:49
(Adds details on launch, background)
March 24 (Reuters) - The U.S. Food and Drug
Administration (FDA) on Friday approved Dutch firm Pharming
Group's PHAR.O 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)
((Mariam.ESunny@thomsonreuters.com))