Pharming Group received a Complete Response Letter from the FDA for its supplemental New Drug Application for Joenja, an oral treatment for children with APDS. The FDA raised concerns about underexposure in lower weight pediatric patients and requested additional data on dosing and batch testing. Pharming plans to address these issues and work with the FDA on next steps. Joenja is currently approved for patients 12 and older. The treatment showed positive results in a Phase III study for children aged 4 to 11, demonstrating improvements in APDS symptoms. The FDA granted Priority Review in October 2025 due to the lack of approved treatments for children under 12 with APDS globally.

Read more at GlobeNewswire: Pharming Group receives Complete Response Letter from U.S.