FDA approves Pharming’s Joenja® as first treatment for children with APDS in the U.S.
Pharming won FDA approval of its supplemental New Drug Application for Joenja (leniolisib) at 40 mg and 50 mg twice-daily dosing for children aged 4 to 11 years weighing at least 27 kg with APDS, making it the first FDA-approved treatment for this pediatric population in the U.S. The newly approved doses are expected to be available to eligible U.S. patients in October through Pharming's specialty distribution network, and a separate sNDA covering children weighing 13 kg to less than 27 kg was submitted on July 30.
Key figures
- Drug Name
- Joenja (leniolisib)
- Phase Of Trial
- Phase III
- Pending snda
- lower doses for children weighing 13 kg to less than 27 kg, submitted July 30
- Min weight kg
- 27
- Apds prevalence
- approximately 1 to 2 people per million worldwide
- Approved dosing
- 40 mg and 50 mg twice-daily
- Us availability
- October 2026
- Pediatric age range
- 4 to 11 years
- Trial duration weeks
- 12
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