{"success":true,"data":{"pressRelease":{"id":"141765","rtpr_id":"nGNXh2Cs0-20260911","ticker":"PHAR","exchange":"Euronext Amsterdam","all_tickers":["PHAR"],"title":"FDA approves Pharming’s Joenja® as first treatment for children with APDS in the U.S.","author":"Globe Newswire","published_at":"2026-09-11T17:59:59.465Z","article_body":"* FDA approval expands Joenja indication to include children with activated\nphosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) ages 4 to 11 who\nweigh at least 27kg\n* Newly approved Joenja doses are expected to be available in October\nLeiden, the Netherlands, September 11, 2026: Pharming (Euronext Amsterdam:\nPHARM/Nasdaq: PHAR), a global biotechnology company focused on rare immune\nand genetic diseases, today announced that the U.S. Food and Drug\nAdministration (FDA) has approved its supplemental New Drug Application (sNDA)\nfor 40 mg and 50 mg twice-daily dosing of Joenja® (leniolisib), an oral,\nselective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a treatment\nfor children aged 4 to 11 years weighing at least 27 kg with activated\nphosphoinositide 3-kinase delta syndrome (APDS), a rare primary\nimmunodeficiency. With this approval, Joenja becomes the first FDA-approved\ntreatment for children aged 4 to 11 years with APDS in the U.S. The newly\napproved Joenja doses are expected to be available to eligible pediatric\npatients in the U.S. in October through Pharming’s established specialty\ndistribution network and patient-support infrastructure.\n\nOn July 30, Pharming also submitted a separate sNDA seeking approval of lower\nJoenja doses for pediatric APDS patients aged 4 to11 years who weigh 13 kg to\nless than 27 kg.\n\n“To give children living with APDS the best chance at a healthier future,\nearly disease intervention is critical,” commented Eveline Wu, M.D., MSCR,\nAssociate Professor of Pediatrics at the University of North Carolina at\nChapel Hill. “Previously, our best approach for children aged 4 to 11 years\nwith APDS was to provide an accurate diagnosis and a treatment plan to manage\nthe symptoms of APDS. With the approval of Joenja for this pediatric\npopulation, we can now take that support even further to address the\nunderlying pathway of disease, with the goal of reducing symptoms and\npreventing the irreversible complications that often arise with APDS.”\n\n“APDS is not a disease that waits. It can affect important aspects of\nchildhood, potentially impacting time in school, time with friends, and other\nactivities of daily life. This approval is a meaningful step forward because\nit gives younger patients and their physicians another much-needed option\nearlier in the disease journey,” said Vanessa Tenembaum, CEO of the Jeffrey\nModell Foundation, an international, non-profit, organization dedicated to\nhelping individuals and family members affected by primary immunodeficiency\ndisorders. “For families living with APDS, it means more than progress; it\nmeans renewed hope, greater possibility and a clearer path forward.”\n\nAPDS typically manifests in early childhood and is characterized by\nsignificant immune dysregulation and recurrent sinopulmonary infections, which\nover time can lead to permanent lung damage and other serious complications.\nFor children living with APDS, the burden can extend beyond clinical symptoms.\nRecurrent infections, medical appointments and treatment demands may disrupt\nschool attendance, learning and social development, adding to the daily impact\non patients and families.\n\n“Today’s approval marks an important achievement in our efforts to expand\nthe availability of Joenja to more individuals living with APDS. We are\ngrateful to the FDA, trial participants, caregivers, investigators, healthcare\nproviders and the APDS community whose partnership helped make this milestone\npossible,” said Leverne Marsh, Chief Commercial Officer of Pharming. “Our\nimmediate priority is to support physicians and families in navigating the\naccess pathway so treatment can start as quickly and responsibly as\npossible.”\n\nJoenja received approval from the U.S. FDA for the treatment of APDS in adult\nand pediatric patients 12 years of age and older in March 2023. Today’s\napproval is supported by data from a multinational, open-label, single-arm\nPhase III study in children aged 4 to 11 years with APDS. The study\ndemonstrated improvements over 12 weeks in two clinically relevant hallmarks\nof the condition, reduced lymphadenopathy and increased naïve B cells,\ntogether indicating an improvement in the underlying immune defect. The safety\nprofile was consistent with prior Joenja experience; all treatment emergent\nadverse events were mild to moderate in nature, and no drug related serious\nadverse events were observed.\n\nUS Important Safety Information for Joenja® (leniolisib)\n\nINDICATIONS AND USAGE \nJoenja® (leniolisib) is a kinase inhibitor indicated for the treatment of\nactivated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult\nand pediatric patients 4 years of age and older who weigh 27 kg or greater.\n\nIMPORTANT SAFETY INFORMATION \nVerify pregnancy status in females of reproductive potential prior to\ninitiating treatment with Joenja.\n\nJoenja may cause fetal harm when administered to a pregnant woman. Advise\npatients of the potential risk to a fetus and to use highly effective methods\nof contraception during treatment with Joenja and for 1 week after the last\ndose. Additionally, advise women not to breastfeed during treatment with\nJoenja and for 1 week after the last dose.\n\nLive, attenuated vaccinations may be less effective if administered during\nJoenja treatment.\n\nJoenja may cause hypersensitivity reaction(s), including anaphylaxis. Advise\npatients to discontinue Joenja and to seek immediate medical attention if they\ndevelop any signs and symptoms of serious allergic reactions.\n\nUse of Joenja in patients with moderate to severe hepatic impairment is not\nrecommended. There is no recommended dosage for patients under 4 years of age\nor weighing less than 27 kg.\n\nAvoid co-administration of Joenja with other medications known to be strong\nCYP3A4 inhibitors, strong or moderate CYP3A4 inducers, or BCRP, OATP1B1, and\nOATP1B3 substrates.\n\nMost common adverse reaction (incidence >10%):\n* Adult and pediatric patients 12 years of age and older: headache, sinusitis,\natopic dermatitis, and weight gain.\n* Pediatric patients 4 to <12 years of age who weigh 27 kg or greater:\nabdominal pain, cough, and respiratory tract infection.\nSeven (33%) patients 12 years and older (n=21) and 5 (63%) patients 4 to 12\nyears of age and weighing more than 27 kg or greater (n=8) developed an\nabsolute neutrophil count (ANC) between 500 and 1500 cells/microL after\nreceiving Joenja. No patients developed an ANC <500 cells/microL and there\nwere no reports of infection associated with neutropenia.\n\nBefore prescribing Joenja, please read the full Prescribing Information.\n\nJoenja is available in 40 mg, 50 mg, and 70 mg tablets.\n\nBased on PI Approved: 09/2026\n\nAbout Activated Phosphoinositide 3-Kinase δ Syndrome (APDS) \nAPDS is a rare primary immunodeficiency that was first characterized in 2013.\nAPDS is caused by variants in either one of two identified genes known\nas PIK3CD or PIK3R1, which are vital to the development and function of\nimmune cells in the body. Variants of these genes lead to hyperactivity of the\nPI3Kδ (phosphoinositide 3-kinase delta) pathway, which causes immune cells to\nfail to mature and function properly, leading to immunodeficiency and\ndysregulation(1,2,3) APDS is characterized by a variety of symptoms,\nincluding severe, recurrent sinopulmonary infections, lymphoproliferation,\nautoimmunity, and enteropathy.(4,5) Because these symptoms can be associated\nwith a variety of conditions, including other primary immunodeficiencies, it\nhas been reported that people with APDS are frequently misdiagnosed and suffer\na median 7-year diagnostic delay.(6 )As APDS is a progressive disease, this\ndelay may lead to an accumulation of damage over time, including permanent\nlung damage and lymphoma.(4-7) A definitive diagnosis can be made through\ngenetic testing. APDS affects approximately 1 to 2 people per million\nworldwide.(8) \n\nAbout Joenja®\nJoenja® (leniolisib) is an oral small molecule phosphoinositide 3-kinase\ndelta (PI3Kẟ) inhibitor approved as the first and only treatment of\nactivated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in the\nU.S., U.K., Australia, Israel, Canada and the European Union in adult and\npediatric patients 12 years of age and older and in Japan for patients 4 years\nof age and older. Joenja® inhibits the production of\nphosphatidylinositol-3-4-5-trisphosphate, which serves as an important\ncellular messenger and regulates a multitude of cell functions such as\nproliferation, differentiation, cytokine production, cell survival,\nangiogenesis, and metabolism. Results from a randomized, placebo-controlled\nPhase III clinical trial demonstrated statistically significant improvement in\nthe coprimary endpoints, reflecting a favorable impact on the immune\ndysregulation and deficiency seen in these patients, and open label extension\ndata has supported the safety and tolerability of\nlong-term leniolisib administration.(9,10)   \nLeniolisib is currently under regulatory review for the treatment of APDS in\nCanada and several other countries. Leniolisib is also being evaluated in\ntwo Phase II clinical trials in primary immunodeficiencies (PIDs) with immune\ndysregulation. The safety and efficacy of leniolisib has not\nbeen established for PIDs with immune dysregulation beyond APDS.  \n\nAbout Pharming\nPharming Group N.V. (Euronext Amsterdam: PHARM/Nasdaq: PHAR) is a global\nbiotechnology company that develops and commercializes innovative\nmedicines for people living with rare immune and genetic diseases.\n\nWe combine specialized scientific, medical, regulatory and\ncommercial expertise to advance a focused portfolio of approved medicines\nand development programs that address significant unmet medical\nneeds. Guided by insights from patients and the wider rare disease\ncommunity, we are dedicated to delivering innovative therapies for some of\nthe most challenging rare diseases. \n\nPharming is headquartered in Leiden, the Netherlands, with operations in the\nUnited States and Europe. \n\nFor more information, visit www.pharming.com and follow us on LinkedIn\n(https://www.globenewswire.com/Tracker?data=gAI0RoVDcIGJT9OjzbymRm4sYumy7QaTkObuc6RAeAnQqgzkGS6Ox6MxZEBAWXs0JmLO2NGd-Vd27KObxOz4DjzoDIOByaseHPu-CZwJemw=). \n\nForward-looking Statements \nThis press release may contain forward-looking statements. Forward-looking\nstatements are statements of future expectations that are based on\nmanagement’s current expectations and assumptions and involve known and\nunknown risks and uncertainties that could cause actual results, performance,\nor events to differ materially from those expressed or implied in these\nstatements. These forward-looking statements are identified by their use of\nterms and phrases such as “aim”, “ambition”, ‘‘anticipate’’,\n‘‘believe’’, ‘‘could’’, ‘‘estimate’’,\n‘‘expect’’, ‘‘goals’’, ‘‘intend’’, ‘‘may’’,\n“milestones”, ‘‘objectives’’, ‘‘outlook’’,\n‘‘plan’’, ‘‘probably’’, ‘‘project’’,\n‘‘risks’’, “schedule”, ‘‘seek’’, ‘‘should’’,\n‘‘target’’, ‘‘will’’ and similar terms and phrases. Examples\nof forward-looking statements may include statements with respect to timing\nand progress of Pharming's preclinical studies and clinical trials of its\nproduct candidates, Pharming's clinical and commercial prospects, and\nPharming's expectations regarding its projected working capital requirements\nand cash resources, which statements are subject to a number of risks,\nuncertainties and assumptions, including, but not limited to the scope,\nprogress and expansion of Pharming's clinical trials and ramifications for the\ncost thereof; and clinical, scientific, regulatory, commercial, competitive\nand technical developments. In light of these risks and uncertainties, and\nother risks and uncertainties that are described in Pharming's 2025 Annual\nReport and the Annual Report on Form 20-F for the year ended December 31,\n2025, filed with the U.S. Securities and Exchange Commission, the events and\ncircumstances discussed in such forward-looking statements may not occur, and\nPharming's actual results could differ materially and adversely from those\nanticipated or implied thereby. All forward-looking statements contained in\nthis press release are expressly qualified in their entirety by the cautionary\nstatements contained or referred to in this section. Readers should not place\nundue reliance on forward-looking statements. Any forward-looking statements\nspeak only as of the date of this press release and are based on information\navailable to Pharming as of the date of this release. Pharming does not\nundertake any obligation to publicly update or revise any forward-looking\nstatement as a result of new information, future events or other information.\n\nInside Information\nThis press release relates to the disclosure of information that qualifies, or\nmay have qualified, as inside information within the meaning of Article 7(1)\nof the EU Market Abuse Regulation.\n\nReferences \n1. FDA. Priority Review. Available at:\nhttps://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/priority-review\nAccessed October 2025. \n2. Lucas CL, et al. Nat Immunol. 2014;15(1):88-97.\n3. Elkaim E, et al. J Allergy Clin Immunol. 2016;138(1):210-218.\n4. Nunes-Santos C, Uzel G, Rosenzweig SD. J Allergy Clin Immunol.\n2019;143(5):1676-1687. \n5. Coulter TI, et al. J Allergy Clin Immunol. 2017;139(2):597-606.\n6. Maccari ME, et al. Front Immunol. 2018;9:543.\n7. Jamee M, et al. Clin Rev Allergy Immunol. 2020 Dec;59(3):323-333.\n8. Condliffe AM, Chandra A. Front Immunol. 2018;9:338.\n9. Rao VK, et al Blood. 2023 Mar 2;141(9):971-983.\n10. Rao VK, et al.  J Allergy Clin Immunol 2024;153:265-74.\nFor further public information, contact:\nPharming\nMichael Levitan, VP Investor Relations & Capital Markets\nT: +1 (908) 705 1696\nE: investor@pharming.com\n\nSaskia Mehring, Head of Corporate Communications\nT: +31 6 28 32 60 41\nE: media.relations@pharming.com\n\nMedia Relations\nJulia Deutsch (Lyra Strategic Advisory on behalf of Pharming)\nE:  JDeutsch@lyraadvisory.com\n\nNetherlands: Leon Melens (LifeSpring Life Sciences Communication on behalf of\nPharming)\nT: +31 6 53 81 64 27\n\nAttachment\n*     FDA approves Pharming’s Joenja as first treatment for children with\nAPDS in the US_EN_11SEP26\n(https://ml-eu.globenewswire.com/Resource/Download/d4526c5c-8a20-40b3-88cf-0c4b0009d64d)\n(https://www.globenewswire.com/NewsRoom/AttachmentNg/f164672f-d67c-41d2-9c79-46caee419077)\n\n\n\nGlobeNewswire, Inc. 2026","article_body_html":"","raw_payload":{"data":{"id":"nGNXh2Cs0-20260911","title":"FDA approves Pharming’s Joenja® as first treatment for children with APDS in the U.S.","author":"Globe Newswire","ticker":"PHAR","created":"2026-09-11T17:59:59.465Z","tickers":["PHAR"],"exchange":"Euronext Amsterdam","article_body":"* FDA approval expands Joenja indication to include children with activated\nphosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) ages 4 to 11 who\nweigh at least 27kg\n* Newly approved Joenja doses are expected to be available in October\nLeiden, the Netherlands, September 11, 2026: Pharming (Euronext Amsterdam:\nPHARM/Nasdaq: PHAR), a global biotechnology company focused on rare immune\nand genetic diseases, today announced that the U.S. Food and Drug\nAdministration (FDA) has approved its supplemental New Drug Application (sNDA)\nfor 40 mg and 50 mg twice-daily dosing of Joenja® (leniolisib), an oral,\nselective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a treatment\nfor children aged 4 to 11 years weighing at least 27 kg with activated\nphosphoinositide 3-kinase delta syndrome (APDS), a rare primary\nimmunodeficiency. With this approval, Joenja becomes the first FDA-approved\ntreatment for children aged 4 to 11 years with APDS in the U.S. The newly\napproved Joenja doses are expected to be available to eligible pediatric\npatients in the U.S. in October through Pharming’s established specialty\ndistribution network and patient-support infrastructure.\n\nOn July 30, Pharming also submitted a separate sNDA seeking approval of lower\nJoenja doses for pediatric APDS patients aged 4 to11 years who weigh 13 kg to\nless than 27 kg.\n\n“To give children living with APDS the best chance at a healthier future,\nearly disease intervention is critical,” commented Eveline Wu, M.D., MSCR,\nAssociate Professor of Pediatrics at the University of North Carolina at\nChapel Hill. “Previously, our best approach for children aged 4 to 11 years\nwith APDS was to provide an accurate diagnosis and a treatment plan to manage\nthe symptoms of APDS. With the approval of Joenja for this pediatric\npopulation, we can now take that support even further to address the\nunderlying pathway of disease, with the goal of reducing symptoms and\npreventing the irreversible complications that often arise with APDS.”\n\n“APDS is not a disease that waits. It can affect important aspects of\nchildhood, potentially impacting time in school, time with friends, and other\nactivities of daily life. This approval is a meaningful step forward because\nit gives younger patients and their physicians another much-needed option\nearlier in the disease journey,” said Vanessa Tenembaum, CEO of the Jeffrey\nModell Foundation, an international, non-profit, organization dedicated to\nhelping individuals and family members affected by primary immunodeficiency\ndisorders. “For families living with APDS, it means more than progress; it\nmeans renewed hope, greater possibility and a clearer path forward.”\n\nAPDS typically manifests in early childhood and is characterized by\nsignificant immune dysregulation and recurrent sinopulmonary infections, which\nover time can lead to permanent lung damage and other serious complications.\nFor children living with APDS, the burden can extend beyond clinical symptoms.\nRecurrent infections, medical appointments and treatment demands may disrupt\nschool attendance, learning and social development, adding to the daily impact\non patients and families.\n\n“Today’s approval marks an important achievement in our efforts to expand\nthe availability of Joenja to more individuals living with APDS. We are\ngrateful to the FDA, trial participants, caregivers, investigators, healthcare\nproviders and the APDS community whose partnership helped make this milestone\npossible,” said Leverne Marsh, Chief Commercial Officer of Pharming. “Our\nimmediate priority is to support physicians and families in navigating the\naccess pathway so treatment can start as quickly and responsibly as\npossible.”\n\nJoenja received approval from the U.S. FDA for the treatment of APDS in adult\nand pediatric patients 12 years of age and older in March 2023. Today’s\napproval is supported by data from a multinational, open-label, single-arm\nPhase III study in children aged 4 to 11 years with APDS. The study\ndemonstrated improvements over 12 weeks in two clinically relevant hallmarks\nof the condition, reduced lymphadenopathy and increased naïve B cells,\ntogether indicating an improvement in the underlying immune defect. The safety\nprofile was consistent with prior Joenja experience; all treatment emergent\nadverse events were mild to moderate in nature, and no drug related serious\nadverse events were observed.\n\nUS Important Safety Information for Joenja® (leniolisib)\n\nINDICATIONS AND USAGE \nJoenja® (leniolisib) is a kinase inhibitor indicated for the treatment of\nactivated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult\nand pediatric patients 4 years of age and older who weigh 27 kg or greater.\n\nIMPORTANT SAFETY INFORMATION \nVerify pregnancy status in females of reproductive potential prior to\ninitiating treatment with Joenja.\n\nJoenja may cause fetal harm when administered to a pregnant woman. Advise\npatients of the potential risk to a fetus and to use highly effective methods\nof contraception during treatment with Joenja and for 1 week after the last\ndose. Additionally, advise women not to breastfeed during treatment with\nJoenja and for 1 week after the last dose.\n\nLive, attenuated vaccinations may be less effective if administered during\nJoenja treatment.\n\nJoenja may cause hypersensitivity reaction(s), including anaphylaxis. Advise\npatients to discontinue Joenja and to seek immediate medical attention if they\ndevelop any signs and symptoms of serious allergic reactions.\n\nUse of Joenja in patients with moderate to severe hepatic impairment is not\nrecommended. There is no recommended dosage for patients under 4 years of age\nor weighing less than 27 kg.\n\nAvoid co-administration of Joenja with other medications known to be strong\nCYP3A4 inhibitors, strong or moderate CYP3A4 inducers, or BCRP, OATP1B1, and\nOATP1B3 substrates.\n\nMost common adverse reaction (incidence >10%):\n* Adult and pediatric patients 12 years of age and older: headache, sinusitis,\natopic dermatitis, and weight gain.\n* Pediatric patients 4 to <12 years of age who weigh 27 kg or greater:\nabdominal pain, cough, and respiratory tract infection.\nSeven (33%) patients 12 years and older (n=21) and 5 (63%) patients 4 to 12\nyears of age and weighing more than 27 kg or greater (n=8) developed an\nabsolute neutrophil count (ANC) between 500 and 1500 cells/microL after\nreceiving Joenja. No patients developed an ANC <500 cells/microL and there\nwere no reports of infection associated with neutropenia.\n\nBefore prescribing Joenja, please read the full Prescribing Information.\n\nJoenja is available in 40 mg, 50 mg, and 70 mg tablets.\n\nBased on PI Approved: 09/2026\n\nAbout Activated Phosphoinositide 3-Kinase δ Syndrome (APDS) \nAPDS is a rare primary immunodeficiency that was first characterized in 2013.\nAPDS is caused by variants in either one of two identified genes known\nas PIK3CD or PIK3R1, which are vital to the development and function of\nimmune cells in the body. Variants of these genes lead to hyperactivity of the\nPI3Kδ (phosphoinositide 3-kinase delta) pathway, which causes immune cells to\nfail to mature and function properly, leading to immunodeficiency and\ndysregulation(1,2,3) APDS is characterized by a variety of symptoms,\nincluding severe, recurrent sinopulmonary infections, lymphoproliferation,\nautoimmunity, and enteropathy.(4,5) Because these symptoms can be associated\nwith a variety of conditions, including other primary immunodeficiencies, it\nhas been reported that people with APDS are frequently misdiagnosed and suffer\na median 7-year diagnostic delay.(6 )As APDS is a progressive disease, this\ndelay may lead to an accumulation of damage over time, including permanent\nlung damage and lymphoma.(4-7) A definitive diagnosis can be made through\ngenetic testing. APDS affects approximately 1 to 2 people per million\nworldwide.(8) \n\nAbout Joenja®\nJoenja® (leniolisib) is an oral small molecule phosphoinositide 3-kinase\ndelta (PI3Kẟ) inhibitor approved as the first and only treatment of\nactivated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in the\nU.S., U.K., Australia, Israel, Canada and the European Union in adult and\npediatric patients 12 years of age and older and in Japan for patients 4 years\nof age and older. Joenja® inhibits the production of\nphosphatidylinositol-3-4-5-trisphosphate, which serves as an important\ncellular messenger and regulates a multitude of cell functions such as\nproliferation, differentiation, cytokine production, cell survival,\nangiogenesis, and metabolism. Results from a randomized, placebo-controlled\nPhase III clinical trial demonstrated statistically significant improvement in\nthe coprimary endpoints, reflecting a favorable impact on the immune\ndysregulation and deficiency seen in these patients, and open label extension\ndata has supported the safety and tolerability of\nlong-term leniolisib administration.(9,10)   \nLeniolisib is currently under regulatory review for the treatment of APDS in\nCanada and several other countries. Leniolisib is also being evaluated in\ntwo Phase II clinical trials in primary immunodeficiencies (PIDs) with immune\ndysregulation. The safety and efficacy of leniolisib has not\nbeen established for PIDs with immune dysregulation beyond APDS.  \n\nAbout Pharming\nPharming Group N.V. (Euronext Amsterdam: PHARM/Nasdaq: PHAR) is a global\nbiotechnology company that develops and commercializes innovative\nmedicines for people living with rare immune and genetic diseases.\n\nWe combine specialized scientific, medical, regulatory and\ncommercial expertise to advance a focused portfolio of approved medicines\nand development programs that address significant unmet medical\nneeds. Guided by insights from patients and the wider rare disease\ncommunity, we are dedicated to delivering innovative therapies for some of\nthe most challenging rare diseases. \n\nPharming is headquartered in Leiden, the Netherlands, with operations in the\nUnited States and Europe. \n\nFor more information, visit www.pharming.com and follow us on LinkedIn\n(https://www.globenewswire.com/Tracker?data=gAI0RoVDcIGJT9OjzbymRm4sYumy7QaTkObuc6RAeAnQqgzkGS6Ox6MxZEBAWXs0JmLO2NGd-Vd27KObxOz4DjzoDIOByaseHPu-CZwJemw=). \n\nForward-looking Statements \nThis press release may contain forward-looking statements. Forward-looking\nstatements are statements of future expectations that are based on\nmanagement’s current expectations and assumptions and involve known and\nunknown risks and uncertainties that could cause actual results, performance,\nor events to differ materially from those expressed or implied in these\nstatements. These forward-looking statements are identified by their use of\nterms and phrases such as “aim”, “ambition”, ‘‘anticipate’’,\n‘‘believe’’, ‘‘could’’, ‘‘estimate’’,\n‘‘expect’’, ‘‘goals’’, ‘‘intend’’, ‘‘may’’,\n“milestones”, ‘‘objectives’’, ‘‘outlook’’,\n‘‘plan’’, ‘‘probably’’, ‘‘project’’,\n‘‘risks’’, “schedule”, ‘‘seek’’, ‘‘should’’,\n‘‘target’’, ‘‘will’’ and similar terms and phrases. Examples\nof forward-looking statements may include statements with respect to timing\nand progress of Pharming's preclinical studies and clinical trials of its\nproduct candidates, Pharming's clinical and commercial prospects, and\nPharming's expectations regarding its projected working capital requirements\nand cash resources, which statements are subject to a number of risks,\nuncertainties and assumptions, including, but not limited to the scope,\nprogress and expansion of Pharming's clinical trials and ramifications for the\ncost thereof; and clinical, scientific, regulatory, commercial, competitive\nand technical developments. In light of these risks and uncertainties, and\nother risks and uncertainties that are described in Pharming's 2025 Annual\nReport and the Annual Report on Form 20-F for the year ended December 31,\n2025, filed with the U.S. Securities and Exchange Commission, the events and\ncircumstances discussed in such forward-looking statements may not occur, and\nPharming's actual results could differ materially and adversely from those\nanticipated or implied thereby. All forward-looking statements contained in\nthis press release are expressly qualified in their entirety by the cautionary\nstatements contained or referred to in this section. Readers should not place\nundue reliance on forward-looking statements. Any forward-looking statements\nspeak only as of the date of this press release and are based on information\navailable to Pharming as of the date of this release. Pharming does not\nundertake any obligation to publicly update or revise any forward-looking\nstatement as a result of new information, future events or other information.\n\nInside Information\nThis press release relates to the disclosure of information that qualifies, or\nmay have qualified, as inside information within the meaning of Article 7(1)\nof the EU Market Abuse Regulation.\n\nReferences \n1. FDA. Priority Review. Available at:\nhttps://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/priority-review\nAccessed October 2025. \n2. Lucas CL, et al. Nat Immunol. 2014;15(1):88-97.\n3. Elkaim E, et al. J Allergy Clin Immunol. 2016;138(1):210-218.\n4. Nunes-Santos C, Uzel G, Rosenzweig SD. J Allergy Clin Immunol.\n2019;143(5):1676-1687. \n5. Coulter TI, et al. J Allergy Clin Immunol. 2017;139(2):597-606.\n6. Maccari ME, et al. Front Immunol. 2018;9:543.\n7. Jamee M, et al. Clin Rev Allergy Immunol. 2020 Dec;59(3):323-333.\n8. Condliffe AM, Chandra A. Front Immunol. 2018;9:338.\n9. Rao VK, et al Blood. 2023 Mar 2;141(9):971-983.\n10. Rao VK, et al.  J Allergy Clin Immunol 2024;153:265-74.\nFor further public information, contact:\nPharming\nMichael Levitan, VP Investor Relations & Capital Markets\nT: +1 (908) 705 1696\nE: investor@pharming.com\n\nSaskia Mehring, Head of Corporate Communications\nT: +31 6 28 32 60 41\nE: media.relations@pharming.com\n\nMedia Relations\nJulia Deutsch (Lyra Strategic Advisory on behalf of Pharming)\nE:  JDeutsch@lyraadvisory.com\n\nNetherlands: Leon Melens (LifeSpring Life Sciences Communication on behalf of\nPharming)\nT: +31 6 53 81 64 27\n\nAttachment\n*     FDA approves Pharming’s Joenja as first treatment for children with\nAPDS in the US_EN_11SEP26\n(https://ml-eu.globenewswire.com/Resource/Download/d4526c5c-8a20-40b3-88cf-0c4b0009d64d)\n(https://www.globenewswire.com/NewsRoom/AttachmentNg/f164672f-d67c-41d2-9c79-46caee419077)\n\n\n\nGlobeNewswire, Inc. 2026"},"type":"article","timestamp":"2026-09-11T17:59:59.503955774Z","server_sent_at_ms":1789149599503},"received_at":"2026-09-11T17:59:59.558Z","source_url":null},"analysis":{"id":"130603","press_release_id":"141765","analysis_json":{"industry":{"label":"Biotechnology","sector":"Health Care"},"redFlags":["Ultra-rare disease prevalence (approximately 1-2 people per million worldwide) limits the incremental commercial contribution from the pediatric expansion","Approval covers only patients weighing at least 27 kg; the sNDA for the 13-27 kg group remains pending"],"eventType":"fda_approval","narrative":"Pharming won FDA approval of its supplemental New Drug Application for Joenja (leniolisib) at 40 mg and 50 mg twice-daily dosing for children aged 4 to 11 years weighing at least 27 kg with APDS, making it the first FDA-approved treatment for this pediatric population in the U.S.\n\nThe newly approved doses are expected to be available to eligible U.S. patients in October through Pharming's specialty distribution network, and a separate sNDA covering children weighing 13 kg to less than 27 kg was submitted on July 30.\n\nThe approval is supported by an open-label, single-arm Phase III study showing reduced lymphadenopathy and increased naïve B cells over 12 weeks, with all treatment-emergent adverse events mild to moderate and no drug-related serious adverse events observed.","sentiment":"bullish","agentHooks":{"shouldPost":true,"suggestedAngle":"First FDA-approved APDS treatment for children aged 4-11 widens Joenja's addressable population, with pediatric doses on shelves in October."},"keyFigures":{"drugName":"Joenja (leniolisib)","phaseOfTrial":"Phase III","customDimensions":{"pending_snda":"lower doses for children weighing 13 kg to less than 27 kg, submitted July 30","min_weight_kg":27,"apds_prevalence":"approximately 1 to 2 people per million worldwide","approved_dosing":"40 mg and 50 mg twice-daily","us_availability":"October 2026","pediatric_age_range":"4 to 11 years","trial_duration_weeks":12}},"quotedText":"Today’s approval marks an important achievement in our efforts to expand\nthe availability of Joenja to more individuals living with APDS.","namedEntities":{"people":[{"name":"Eveline Wu, M.D., MSCR","role":"Associate Professor of Pediatrics, University of North Carolina at Chapel Hill"},{"name":"Vanessa Tenembaum","role":"CEO, Jeffrey Modell Foundation"},{"name":"Leverne Marsh","role":"Chief Commercial Officer, Pharming"}],"products":["Joenja (leniolisib)"],"companies":[{"name":"Pharming Group N.V.","ticker":"PHAR","relationship":"filer"},{"name":"U.S. Food and Drug Administration","relationship":"regulator"},{"name":"Jeffrey Modell Foundation","relationship":"patient advocacy organization"}],"dollarAmounts":[]},"materialImpact":{"score":4,"reasoning":"FDA approved Pharming's sNDA making Joenja the first FDA-approved treatment for children aged 4 to 11 with APDS, expanding the label beyond the existing 12+ approval with a near-term commercial path (October availability). A meaningful regulatory win for the filer, though incremental to an already-approved franchise in an ultra-rare disease."},"tickerRelevance":{"others":[{"ticker":"PHARM","relevance":"Euronext Amsterdam listing of the same issuer"}],"primary":"PHAR"},"globalImportance":35,"audienceRelevance":25,"eventTypeSecondary":["product_launch"],"importanceComponents":{"tickerTier":"small-cap biotech","eventGravity":"FDA sNDA label expansion (pediatric 4-11)","sectorWeight":"health care / regulatory","issuerAuthored":true,"firstMoverAngle":"first FDA-approved APDS treatment for ages 4-11","marketScopeLimit":"ultra-rare prevalence caps commercial upside"}},"event_type":"fda_approval","event_type_secondary":["product_launch"],"sentiment":"bullish","material_impact_score":4,"narrative":"Pharming won FDA approval of its supplemental New Drug Application for Joenja (leniolisib) at 40 mg and 50 mg twice-daily dosing for children aged 4 to 11 years weighing at least 27 kg with APDS, making it the first FDA-approved treatment for this pediatric population in the U.S.\n\nThe newly approved doses are expected to be available to eligible U.S. patients in October through Pharming's specialty distribution network, and a separate sNDA covering children weighing 13 kg to less than 27 kg was submitted on July 30.\n\nThe approval is supported by an open-label, single-arm Phase III study showing reduced lymphadenopathy and increased naïve B cells over 12 weeks, with all treatment-emergent adverse events mild to moderate and no drug-related serious adverse events observed.","key_figures":{"drugName":"Joenja (leniolisib)","phaseOfTrial":"Phase III","customDimensions":{"pending_snda":"lower doses for children weighing 13 kg to less than 27 kg, submitted July 30","min_weight_kg":27,"apds_prevalence":"approximately 1 to 2 people per million worldwide","approved_dosing":"40 mg and 50 mg twice-daily","us_availability":"October 2026","pediatric_age_range":"4 to 11 years","trial_duration_weeks":12}},"named_entities":{"people":[{"name":"Eveline Wu, M.D., MSCR","role":"Associate Professor of Pediatrics, University of North Carolina at Chapel Hill"},{"name":"Vanessa Tenembaum","role":"CEO, Jeffrey Modell Foundation"},{"name":"Leverne Marsh","role":"Chief Commercial Officer, Pharming"}],"products":["Joenja (leniolisib)"],"companies":[{"name":"Pharming Group N.V.","ticker":"PHAR","relationship":"filer"},{"name":"U.S. Food and Drug Administration","relationship":"regulator"},{"name":"Jeffrey Modell Foundation","relationship":"patient advocacy organization"}],"dollarAmounts":[]},"model_name":"glm-5.3-flash","prompt_hash":"sha256:727b4b9429a443af","schema_hash":"sha256:05005c02d9cffac9","created_at":"2026-09-11T18:00:37.469Z","global_importance":35,"audience_relevance":25,"importance_components":{"tickerTier":"small-cap biotech","eventGravity":"FDA sNDA label expansion (pediatric 4-11)","sectorWeight":"health care / regulatory","issuerAuthored":true,"firstMoverAngle":"first FDA-approved APDS treatment for ages 4-11","marketScopeLimit":"ultra-rare prevalence caps commercial upside"}},"durationMs":37899,"modelName":"glm-5.3-flash"}}