The U.S. FDA approved a supplemental New Drug Application for Joenja (leniolisib), expanding its indication to treat children aged 4 to 11 years weighing at least 27 kg with activated phosphoinositide 3-kinase delta syndrome (APDS). This approval establishes Joenja as the first FDA-approved treatment for this pediatric population. Newly approved doses are expected to be available in October through Pharming’s specialty distribution network. A separate submission for lower doses for patients weighing 13 kg to less than 27 kg was filed on July 30, 2026. The approval is based on Phase III study data demonstrating improvements in lymphadenopathy and naïve B cells. Pharming Group N.V. is a global biotechnology company that develops and commercializes innovative medicines for rare immune and genetic diseases.

Pharming Group N.V. announced the commercial launch of Joenja (leniolisib) in Japan for adult and pediatric patients with activated PI3K delta syndrome (APDS) aged 4 years and older, following its inclusion on the Japan National Health Insurance drug price list. This marks the first commercial availability of Joenja for children aged 4 to 11 worldwide. OrphanPacific, Inc. serves as the Marketing Authorization holder responsible for supply and distribution in Japan under an agreement with Pharming. The launch follows approval by Japan’s Ministry of Health, Labour and Welfare (MHLW) in March 2026. Leverne Marsh, Chief Commercial Officer, described this as a significant milestone in global expansion addressing unmet needs in rare disease communities. Pharming Group N.V. is a global biopharmaceutical company dedicated to developing and commercializing innovative medicines for rare, debilitating, and life-threatening diseases.

Pharming Group N.V. reported second quarter 2026 total revenues of US$90.2 million, a 3% decrease from the prior year period. RUCONEST revenue fell 10% to US$72.3 million due to market dynamics and inventory drawdowns, while Joenja (leniolisib) revenue surged 40% to US$17.9 million driven by U.S. growth and a new European launch in Germany. Operating profit declined to US$1.3 million from US$10.8 million, impacted by US$4.9 million in manufacturing-related inventory impairments and costs associated with closing its Évry, France production site. The company revised its 2026 total revenue guidance down by US$30 million to a range of US$375 million–US$395 million but improved operating expense guidance by US$15 million to US$315 million–US$320 million. Pharming anticipates near-term clinical readouts for leniolisib in CVID patients in the fourth quarter 2026 and expects RUCONEST revenues to stabilize in the second half of the year. Pharming Group N.V. is a global biopharmaceutical company developing and commercializing innovative therapies for rare, debilitating, and life-threatening diseases.

Pharming Group N.V. announced that the U.S. FDA has accepted its resubmitted supplemental New Drug Application (sNDA) for Joenja® (leniolisib) to treat children aged 4 to 11 years with APDS. The resubmission seeks approval for 40 mg and 50 mg twice-daily dosing for pediatric patients weighing 27 kg or more, with a PDUFA target action date of October 24, 2026.