PHAR Pharming Group

$PHAR The FDA has accepted Pharming’s supplemental New Drug Application seeking approval for lower doses of Joenja® for children aged 4 years and older with APDS who weigh between 13 kg and 27 kg. The application has been granted Priority Review, with a PDUFA target action date of January 30, 2027. This follows the recent U.S. approval of Joenja for children aged 4 to 11 weighing at least 27 kg. If approved, Joenja would be available to eligible patients with APDS aged 4 years and older who weigh 13 kg or more, extending treatment access to lower-weight children who are not covered by the current U.S. indication. The application is supported by positive Phase III data in children aged 4 to 11, which showed improvements in two clinically relevant hallmarks of APDS: reduced lymphadenopathy and increased naïve B cells over 12 weeks. Read the full announcement: Our News | Pharming Group N.V. | Pharming

$PHAR Pharming has announced positive topline results from its Phase II study of leniolisib in patients with primary immunodeficiencies (PIDs) involving PI3Kδ-driven immune dysregulation. The findings showed a generally favorable tolerability profile and clinical improvements across multiple measures of immune dysregulation, including lymphoproliferative disease burden, with a mean spleen volume reduction (SVR) of 26.4% and reductions in index lesions. The results have been accepted as a late-breaking abstract at ESID 2026 and provide encouraging early support for expanding leniolisib beyond APDS into potentially larger patient populations. Additional findings to be shared at ESID, followed by topline results from the separate Phase II study of leniolisib in CVID expected in the fourth quarter, will provide further insight into the potential breadth of the leniolisib opportunity. Read the full announcement: Our News | Pharming Group N.V. | Pharming

$PHAR The FDA has approved Joenja® for children ages 4 to 11 with APDS who weigh at least 27 kg, ahead of the October PDUFA date. Joenja is now the first FDA-approved treatment for this pediatric APDS population in the U.S. The newly approved doses are expected to become available in October through Pharming’s existing U.S. distribution and patient-support infrastructure. This approval represents another important step in expanding Joenja’s reach to younger patients and supporting its continued growth. A separate application for children ages 4 to 11 weighing between 13 kg and less than 27 kg was submitted to the FDA on July 30 and is under review. Pharming has identified ~60 eligible patients in total between 4 to 11 in the US, representing an attractive revenue opportunity.

$PHAR Another Pharming PR narrative that sounds broader than the actual approval. Joenja is now approved for children 4–11, but only if they weigh at least 27 kg. That weight corresponds roughly to the average weight of a 9-year-old, meaning a significant part of the 4–11 age group is still excluded. Yet the headline suggests a full 4–11 approval. Is this transparency, or simply another attempt to make the news sound bigger than it is?

$PHAR Pharming FDA approves Pharming's Joenja® as first treatment for children with APDS in the U.S. Leiden, the Netherlands, September 11, 2026: Pharming (Euronext Amsterdam: PHARM/Nasdaq: PHAR), a global biotechnology company focused on rare immune and genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved its supplemental New Drug Application (sNDA) for 40 mg and 50 mg twice-daily dosing of Joenja® (leniolisib), an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a treatment for children aged 4 to 11 years weighing at least 27 kg with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency

$PHAR Pharming IPO: $20. Today: $10. , without the CEO there’s apparently nothing left but the same old narrative. No answers to critical questions, just more PR. Stop pushing narratives and start giving investors actual answers.

$PHAR Good news, Pharming wants the best and stragety and Mr courage wasn't good enough. Good decision

$PHAR Pharming is again highlighting “positive” topline results, but investors should separate the data from the narrative. The Phase II PID study involved only 13 patients and was single-arm/open-label. A mean 26.4% reduction in spleen volume is an interesting signal, but it does not establish efficacy in a large new commercial population. Pharming immediately points toward “potentially larger patient populations,” while the separate CVID Phase II results are still expected in Q4. So yes, encouraging early data — but the expansion story remains unproven. The commercial opportunity is still a potential future outcome, not a demonstrated result.

$PHAR FDA approved expanded use of Joenja for children ages 4–11 with APDS. First FDA-approved treatment for this pediatric APDS group in the U.S. Fresh FDA catalyst worth watching Monday.

$PHAR Joehoe Monitor, gelezen? Dit mooie nieuws kwam tegen je verwachting in zeker en ook nog eens eerder. Wat een zege voor al die kinderen die met deze ziekte kampen hè.

$PHAR Pharming wins approval of Joenja for younger age group. The US FDA has approved Pharming Group's (PHAR) Joenja (leniolisib) for children between the ages of four and 11 with a kind of rare genetic immune system disorder.