Pharming Group (PHAR), a biopharmaceutical company, announced that the FDA has declined to approve the supplemental New Drug Application for Joenja as a treatment for children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome, or APDS. The drug is already approved for the treatment of APDS in adult and pediatric patients 12 years of age and older.
Activated phosphoinositide 3-kinase delta syndrome (APDS) is a rare primary immunodeficiency caused by variants in either one of two identified genes known as PIK3CD or PIK3R1, which are vital to the development and function of immune cells in the body.
In its complete response letter for the expanded use of Joenja in children aged 4 to 11 years, the FDA has raised concerns about potential underexposure in lower-weight paediatric patients. So, the FDA has requested additional paediatric pharmacokinetic data to reassess the proposed paediatric doses and confirm that children in the lower-weight dose groups can achieve exposure levels comparable to those in the approved adult and adolescent regimen.
The company believes it can address the clinical pharmacology and batch-testing methodology issues outlined in the letter by working closely with the FDA.
Pharming Group will also determine the next steps for resubmission and has plans to request a Type A meeting with the FDA.
Joenja has been marketed in the U.S. for the treatment of APDS in adult and pediatric patients 12 years of age and older since 2023. The drug generated revenue of $15.1 million in the third quarter of 2025, an increase of 35% compared to third quarter 2024.
Over the year, PHAR has traded between $7.50 and $21.34. The stock closed Friday's after-hours trade at $20.87, up 1.95%.
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