Pharming shares jump 6% as FDA grants priority review for Joenja in children

Investing.com -- Shares of Pharming Group NV (AS:PHAR) rose more than 6% on Wednesday after the FDA granted Priority Review for its supplemental New Drug Application for Joenja (leniolisib) to treat children with Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS) aged 4 to 11 years.

The U.S. Food and Drug Administration set a Prescription Drug User Fee Act (PDUFA) target date of January 31, 2026, advancing the biopharmaceutical company’s prior guidance for a decision in the first half of 2026. 

The FDA decision follows positive results from Pharming’s Phase 3 study announced in December 2024. If approved, Joenja would be the first and only treatment indicated for children with APDS in this age group.

Pharming has identified more than 50 APDS patients in the United States between the ages of 4 and 11 who could be eligible for treatment once approved. 

That represents about one-quarter of all APDS patients currently identified in the U.S., and many are already receiving Joenja through various access programs.

At the end of June, Pharming reported that 114 patients in the U.S. aged 12 and older were on paid therapy, generating $23 million in Joenja revenues in the first half of 2025.

RBC Capital Markets forecasts 2030 estimated Joenja sales of $158 million for APDS and $186 million in total, including risk-adjusted revenues from new indications. 

The brokerage noted that at €0.66 per share, Joenja accounts for roughly 25% of Pharming’s sum-of-the-parts valuation.

In its research note, RBC Capital Markets assigned Pharming a price target of €2.45, maintaining an “outperform” rating with a “speculative risk” designation. 

The analysts emphasized the potential significance of the label expansion, stating that approval “should further unlock Joenja’s treatable patient population.”