Pharming Group says FDA accepts lower-dose Joenja sNDA for review
The application seeks dosing for smaller children with APDS, and the FDA set Jan. 30, 2027 as its target action date.
Adrian Kessler
Children with APDS weighing as little as 13 kilograms could gain access to Joenja after Pharming Group N.V. AMSPHARM NASDAQPHAR said Friday the FDA accepted its lower-dose application for priority review, with a Jan. 30, 2027 target action date.
The supplemental New Drug Application seeks approval of lower doses for children aged four and older who weigh between 13 and 27 kilograms. Acceptance begins the U.S. Food and Drug Administration's review; it does not expand the current label.
Leiden-based Pharming is a small-cap biotechnology company selling medicines for rare immune and genetic diseases in international markets.
Pharming's Nasdaq ADR NASDAQPHAR closed 0.19 per cent higher at US$10.46 on Sept. 25 at 4 p.m. EDT, Google Finance data show. The release lists no publication time, so the company announcement cannot be tied to that trading.
Lower-Weight Children Await a Label Decision
The FDA first approved Joenja for adults and patients aged 12 and older with APDS in March 2023. A September 2026 expansion added children aged four to 11 who weigh at least 27 kilograms, leaving lighter children outside the U.S. label while the new application is reviewed.
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APDS is a genetic primary immunodeficiency linked to changes in either the PIK3CD or PIK3R1 gene. Those changes disrupt immune-cell function and can cause recurrent infections and enlarged lymph nodes. Pharming's Sept. 25 release estimates that one or two people per million worldwide have the condition, but gives no count for the proposed lower-weight U.S. group.
The filing draws on an open-label, single-arm Phase III study of children aged four to 11 and additional clinical pharmacology assessments of the proposed doses. Pharming said that after 12 weeks of treatment, the study showed smaller enlarged lymph nodes and more naive B cells, measures of the underlying immune disorder.
The ClinicalTrials.gov study record, updated May 6, 2026 and checked Sept. 26, lists estimated enrollment of 15 and one leniolisib arm. Pharming's Sept. 25 announcement gave neither the number actually treated nor numerical efficacy results, p values or adverse-event rates, limiting what investors can assess from the release.
"Following the recent approval of Joenja for children aged 4 to 11 years weighing at least 27 kg, this review brings us closer to the possibility of reaching smaller children who currently are ineligible for treatment with Joenja."
Pharming chief medical officer Anurag Relan said this in the Sept. 25 release.
Joenja brought in US$17.9 million in second-quarter revenue, a 40 per cent increase from a year earlier, Pharming reported in its July 30 results. Total company revenue for that quarter was US$90.2 million. The application would extend the eligible weight range for a product already on sale, though the release offered no estimate of added prescriptions.
Rare Immunodeficiency Peers Have Different Labels
X4 Pharmaceuticals, Inc. NASDAQXFOR received FDA approval of Xolremdi for WHIM syndrome in April 2024, another rare primary immunodeficiency. CSL Limited ASXCSL has a broader immunodeficiency product through CSL Behring: Hizentra, a subcutaneous replacement therapy for primary humoral immunodeficiency.
X4 shares NASDAQXFOR closed at US$3.01 on Sept. 25 at 4 p.m. EDT, Google Finance data show. Its WHIM indication and Pharming's APDS label address different diagnoses within the rare immune-disease market.
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The FDA's Jan. 30, 2027 target action date will decide whether Joenja's U.S. label can extend to eligible children aged four and older who weigh at least 13 kilograms.
