Pharming secures priority review for lower-dose pediatric Joenja

Pharming ($PHAR) said the FDA accepted its supplemental application for a lower-dose Joenja formulation and granted priority review. The proposed expansion addresses patients aged four and older with activated PI3K delta syndrome who weigh between 13 and 27 kilograms. The agency’s target decision date is January 30, 2027, giving the company a defined timetable for the assessment.

The filing is supported by an open-label, single-arm Phase 3 pediatric study evaluating lymph-node enlargement and immune-cell measures over 12 weeks. It is distinct from the earlier authorization covering children weighing at least 27 kilograms. Acceptance does not yet permit use in the newly proposed weight group, and the study design lacks a concurrent randomized comparator.

Sources:

• Pharming — supplemental application and priority review, GlobeNewswire release