India, Sept. 11 -- Pharming Group N.V. (PHARM.AS) said the U.S. Food and Drug Administration has approved Joenja (leniolisib) for children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome, or APDS, who weigh at least 27 kg.
The approval expands Joenja's existing U.S. indication and makes it the first FDA-approved treatment for children aged 4 to 11 with APDS. The newly approved 40 mg and 50 mg twice-daily doses are expected to become available to eligible pediatric patients in the U.S. in October through Pharming's specialty distribution and patient-support network.
The FDA decision was supported by data from a multinational Phase 3 study in children aged 4 to 11 with APDS. Over 12 weeks, treatment demonstrated i...