Pharming Gets FDA Priority Review for Infection-Causing Genetic Disorder Drug For Children Aged 4-11

MT Newswires Live10-01

Pharming Group (PHAR) said Wednesday the US Food and Drug Administration accepted its supplemental new drug application for leniolisib to treat children ages four to 11 with APDS, a genetic disorder, and granted priority review with a Jan. 31, 2026, target decision date.

If approved, leniolisib would be the first treatment indicated for APDS in patients under 12, the company said.

The filing is backed by a phase 3 study showing 12-week improvements in lymphadenopathy and naive B cells, plus eight months of safety data, Pharming said.

Leniolisib, marketed as Joenja in the US, is already approved for APDS in patients 12 and older, the company added.

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