REG-FDA approves Pharming’s Joenja® as first treatment for children with APDS in the U.S.
The FDA approved Pharming's 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 that pediatric population in the U.S. Approval was supported by a multinational, open-label, single-arm Phase III study showing reduced lymphadenopathy and increased naïve B cells over 12 weeks; all treatment-emergent adverse events were mild to moderate and no drug-related serious adverse events were observed. The newly approved doses are expected to reach eligible U.S.
Key figures
- Drug Name
- Joenja (leniolisib)
- Phase Of Trial
- Phase III
- Pending snda
- lower doses for patients aged 4 to 11 weighing 13 kg to less than 27 kg, submitted July 30
- Trial design
- multinational, open-label, single-arm Phase III in children aged 4 to 11; improvements in lymphadenopathy and naïve B cells over 12 weeks
- Min weight kg
- 27
- Prior approval
- March 2023 for adult and pediatric patients 12 years and older
- Apds prevalence
- approximately 1 to 2 people per million worldwide
- Approved dosing
- 40 mg and 50 mg twice-daily
- Pediatric age range
- 4 to 11 years
- Expected availability
- October 2026
- First fda approval for population
- Yes
AI analysis
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