Policy & Regulation
Pharming Group's Joenja receives FDA approval to treat APDS
27 March 2023 -

Pharming Group NV (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR), a Netherlands-based global biopharmaceutical company, announced on Friday that it has received approval from the US Food and Drug Administration (FDA) for the company's Joenja (leniolisib) to treat activated phosphoinositide 3-kinase delta (PI3K delta) syndrome (APDS) in adult and paediatric patients 12 years of age and older.

Joenja, an oral, selective PI3K delta inhibitor, is the first and only treatment approved in the US for APDS, a rare and progressive primary immunodeficiency.

The FDA evaluated the New Drug Application (NDA) for Joenja under priority review and has approved the drug based on findings from a multinational, triple-blind, placebo-controlled, randomised Phase II/III clinical trial, which evaluated efficacy and safety in 31 patients diagnosed with APDS aged 12 years and older.

Joenja is expected to launch in the US in early April and will be available for shipment in mid-April.



Related Headlines