Pharming Group N.V. develops and commercializes medicines for rare and ultra-rare diseases, with news focused on RUCONEST®, Joenja® (leniolisib) for activated phosphoinositide 3-kinase delta syndrome, and the company's rare-disease pipeline. Updates commonly cover clinical studies, market authorizations, commercial launches, licensing economics, product revenue trends, and management presentations at healthcare investor conferences.
The company operates as a global biopharmaceutical issuer listed on Euronext Amsterdam and Nasdaq. Recurring coverage also includes financial results, regulatory interactions in major markets, and development programs involving small molecules, biologics, protein replacement therapies, and precision medicines for immunological and genetic conditions.
Pharming Group (Nasdaq: PHAR) expects third-quarter 2026 revenue of approximately US$117.6 million, up approximately 21% from a year earlier. The preliminary, unaudited figure compares with US$97.3 million in third-quarter 2025. RUCONEST revenue reached approximately US$93.8 million, up 14% year over year and 30% sequentially; Joenja contributed US$23.8 million, up 58% year over year.
Expected operating expenses of approximately US$74.6 million compare with US$74.5 million a year earlier. Pharming narrowed 2026 revenue guidance to US$380 million–US$390 million from US$375 million–US$395 million and lowered operating expense guidance to US$310 million–US$315 million from US$315 million–US$320 million. Full results and a business update move to October 27, 2026, from November 5, following revenue above and operating expenses below analyst consensus.
Pharming (PHAR) presented Phase II topline data for leniolisib showing improvements in genetically identifiable primary immunodeficiencies with immune dysregulation beyond APDS.
The single-arm, open-label study enrolled 13 participants treated twice daily over 20 weeks. Mean spleen volume fell 26.4% from baseline in 11 evaluable participants; lymph-node index lesion size fell 33.7% in four. Lung gas-exchange capacity increased by a mean of 8.0% among seven participants impaired at baseline. Lung disease scores decreased in four of five evaluable participants; liver stiffness improved in two of three with elevated baseline stiffness.
All treatment-related adverse events were Grade 1, with no new safety signals. Two participants had serious events deemed unrelated to leniolisib; one discontinued due to acute hepatitis. All 12 completers entered a three-year extension. Pharming expects separate CVID trial topline results in November.
Pharming (PHAR) announced Fabrice Chouraqui’s immediate departure as CEO and executive director and appointed two interim co-CEOs.
Chief Commercial Officer Leverne Marsh will lead patient-focused operations, while Chief Financial Officer Kenneth Lynard will lead global corporate functions. The board has begun a formal search for a permanent successor.
The board and Chouraqui mutually agreed to the transition after concluding that their views on how to execute Pharming’s strategic priorities were not sufficiently aligned. Pharming said its strategy and pipeline remain unchanged.
Pharming (PHAR) announced FDA acceptance and Priority Review of its lower-dose Joenja application for children with APDS.
The supplemental New Drug Application seeks approval of oral Joenja (leniolisib) for children aged 4 years and older weighing between 13 kg and 27 kg. The FDA assigned a target action date of January 30, 2027. If approved, Joenja would be available to eligible patients aged 4 years and older weighing at least 13 kg. The review follows a September 2026 U.S. approval for children aged 4 to 11 years weighing at least 27 kg.
The application is supported by a multinational, open-label, single-arm Phase III study in children aged 4 to 11 years. Over 12 weeks, the study showed reduced lymphadenopathy, or enlarged lymph nodes, and increased naive B cells, a type of immune cell. FDA acceptance and Priority Review are not approval of the proposed lower doses.
Pharming (PHAR) reported positive Phase II topline data for leniolisib in genetically identifiable primary immunodeficiencies (PIDs) with immune dysregulation linked to PI3Kδ signaling, accepted as a late-breaking abstract at ESID 2026.
The single-arm, open-label, intra-patient dose-escalation study evaluated 13 subjects and showed clinical improvements in lymphoproliferative disease, including a mean 26.4% spleen volume reduction and decreases in index lesion size. Leniolisib was generally well-tolerated, with infections as the most common events and no new safety signals, in line with its known safety profile. Additional data will be presented at the ESID meeting in October 2026 in the Netherlands.
Nine of the 13 patients also had common variable immunodeficiency (CVID), and Pharming expects to report separate Phase II topline results of leniolisib in CVID with immune dysregulation in Q4 2026 to inform potential future registrational plans.
Pharming (PHAR) received U.S. FDA approval of a supplemental New Drug Application on 11 September 2026 for new Joenja® (leniolisib) 40 mg and 50 mg twice-daily doses for children with activated phosphoinositide 3-kinase delta syndrome (APDS) aged 4 to 11 years who weigh at least 27 kg. With this decision, Joenja becomes the first FDA-approved treatment option for APDS in this pediatric age group in the U.S.
The newly approved pediatric doses are expected to be available in October through Pharming’s existing specialty distribution and patient-support network. The expanded indication is supported by a multinational open-label Phase III study in children 4 to 11 years that showed 12‑week improvements in lymphadenopathy and naïve B cells, with only mild to moderate treatment-emergent adverse events and no drug-related serious adverse events. Joenja is now indicated in the U.S. for APDS in adults and pediatric patients 4 years and older who weigh 27 kg or greater.
Pharming Group (PHAR) will participate in two investor conferences in New York in September 2026, including the H.C. Wainwright 28th Annual Global Investment Conference on September 14 and the Morgan Stanley 24th Annual Global Healthcare Conference on September 15.
Chief Executive Officer Fabrice Chouraqui will present at H.C. Wainwright on Monday, September 14 at 09:30 EDT/15:30 CEST. A live webcast and replay of this presentation will be available in the “Events and presentations” and “Our news” sections of Pharming’s website.
Pharming (Nasdaq: PHAR) 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 inclusion on the Japan National Health Insurance drug price list.
Japan becomes the first market where Joenja is commercially available for children aged 4 to 11. The product was approved in March by Japan’s Ministry of Health, Labour and Welfare as the first treatment specifically for APDS. Under an agreement with Pharming, OrphanPacific is the Marketing Authorization Holder in Japan and, in collaboration with Pharming, is responsible for supply and distribution.
Pharming Group (Nasdaq: PHAR) will participate in the Canaccord Genuity 46th Annual Growth Conference in Boston on August 12-13, 2026. VP Investor Relations and Capital Markets Michael Levitan will join a Fireside Chat Q&A on August 12 at 10:00 EDT/16:00 CEST. Investors can arrange one-on-one meetings via Pharming’s Investor Relations team or their Canaccord Genuity representative.
Pharming (PHAR) reported preliminary Q2 2026 revenues of US$90.2 million, down 3% year-on-year, as RUCONEST® sales fell 10% to US$72.3 million while Joenja® (leniolisib) rose 40% to US$17.9 million. Gross profit declined 10% to US$75.7 million, impacted by US$4.9 million manufacturing-related inventory impairments.
Operating profit dropped to US$1.3 million from US$10.8 million, with adjusted operating profit at US$7.9 million. Net profit was US$1.6 million. For H1 2026, revenues decreased 6% to US$162.7 million and the company posted a US$3.6 million operating loss. Cash and marketable securities were US$159.5 million on June 30, 2026. Pharming cut 2026 revenue guidance to US$375–395 million (down US$30 million) and improved operating expense guidance to US$315–320 million. Joenja gained multiple approvals and launches, and leniolisib readouts in broader primary immunodeficiencies, including CVID, are expected in Q4 2026.