Pharming Group N.V. received U.S. FDA approval that makes Joenja the first FDA-approved treatment for children with APDS in the country. The expanded indication gives PHAR investors a defined pediatric use for the oral therapy, while eligible patients are expected to access the newly approved doses through Pharming’s established support and distribution infrastructure.

Key Highlights

  • Joenja is an oral, selective PI3Kδ inhibitor for APDS.
  • Pharming submitted a separate sNDA on July 30 for lower pediatric doses.
  • Newly approved Joenja doses are expected to reach eligible patients through Pharming’s specialty distribution network and support infrastructure.

Pharming Group N.V. (NASDAQ: PHAR) received U.S. FDA approval expanding Joenja’s use to children ages 4 to 11 with activated phosphoinositide 3-kinase Delta syndrome, or APDS, who weigh at least 27 kg.

Joenja Gains Pediatric APDS Indication

The approval covers Joenja, also known as leniolisib, as an oral, selective PI3Kδ inhibitor for pediatric patients with APDS. The company announced the FDA decision on September 11. Pharming submitted a separate supplemental New Drug Application on July 30 seeking approval for lower Joenja doses for another group of pediatric APDS patients. The application is separate from the approval announced on September 11. APDS is described as a rare primary immunodeficiency, and Joenja is intended to address the underlying disease pathway. A quoted physician said early disease intervention is critical for children living with APDS.