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StocksMediumUpdated•Originally published 11 September 2026•Updated 11 September 2026•
2 min read

FDA Expands Joenja Approval to Children Aged 4-11 Weighing at Least 27 kg

Key Facts

1The FDA approval covers children aged 4 to 11 weighing at least 27 kg and 40 mg or 50 mg twice-daily dosing.
2Pharming expects the new doses to become available in the United States in October 2026.
3The phase 3 study showed improvement in two APDS-related markers over 12 weeks.
4Joenja's second-quarter 2026 revenue was $17.9 million, up 40%, with 86% coming from the United States.
5PHAR closed at $10.81 on September 10, 2026, after trading between $10.67 and $10.97.

The U.S. Food and Drug Administration approved Pharming's supplemental application to expand Joenja for activated PI3K delta syndrome, or APDS, to children aged 4 to 11 who weigh at least 27 kg. The approval covers 40 mg and 50 mg doses administered twice daily.

Joenja is now the first U.S.-approved treatment for this pediatric APDS group. Pharming expects the new doses to become available to eligible patients in October 2026 through its specialty distribution and patient-support network.

The approval was supported by a multinational, open-label, single-arm phase 3 study in children aged 4 to 11. After 12 weeks, the study showed reduced lymph-node enlargement and increased naïve B cells, two markers associated with the immune dysfunction caused by APDS.

Under the updated label, Joenja is indicated for adults and children aged 4 and older who weigh at least 27 kg, and is available as 40 mg, 50 mg and 70 mg tablets. The most common adverse reactions in patients aged 4 to under 12 were abdominal pain, cough and respiratory-tract infection; all treatment-emergent events in the study were mild to moderate, with no drug-related serious events reported.

The current approval does not cover children weighing less than 27 kg. Pharming submitted a separate supplemental application on July 30, 2026, seeking approval of lower doses for APDS patients aged 4 to 11 weighing from 13 kg to less than 27 kg.

As of June 30, 2026, Pharming had identified about 60 diagnosed U.S. APDS patients aged 4 to 11. Patients weighing at least 27 kg represent a subset of that group, but the company has not disclosed a precise count of patients meeting the new weight threshold.

Joenja generated $17.9 million of revenue in the second quarter of 2026, up 40% from a year earlier, with the United States accounting for 86% of quarterly Joenja revenue. The expanded eligible population therefore creates a potential growth avenue in the drug's main market, although the realized effect will depend on availability and patient access.

Nasdaq-listed PHAR closed at $10.81 on September 10, 2026, the session before the announcement, after trading between $10.67 and $10.97. The supplied market data did not include post-approval trading.