Pharming expands Joenja approval to younger children with APDS

Pharming ($PHAR) secured FDA approval to use Joenja in children aged four to 11 with activated PI3K delta syndrome who weigh at least 27 kilograms. The expansion adds younger patients to the medicine’s existing indication for older patients. The company expects the new pediatric presentations to become available in October.

The supporting pediatric study was open-label and assessed treatment over 12 weeks, reporting reductions in lymph-node size and improvements in immune-cell measures. Reported adverse events were mild or moderate, with no treatment-related serious adverse events. A separate application for children weighing 13 to less than 27 kilograms remains pending. The new authorization therefore does not yet cover every younger child with the condition.

Sources:

• Pharming — FDA approval announcement, GlobeNewswire