In @jhumimmunity.org, Santiago et al. utilize a large, limited, de-identified dataset to explore characteristics of patients with APDS. Hypertension was found in 28.5%. There was variable usage of leniolisib- and leniolisib-treated patients had lower odds of hypertension. rupress.org/jhi/article/...
October 26, 2026 at 6:30 PM
In @jhumimmunity.org, Santiago et al. utilize a large, limited, de-identified dataset to explore characteristics of patients with APDS. Hypertension was found in 28.5%. There was variable usage of leniolisib- and leniolisib-treated patients had lower odds of hypertension. rupress.org/jhi/article/...
Santiago et al. utilize a large, limited, de-identified dataset to explore characteristics of patients with APDS. Hypertension was found in 28.5% of patients. There was variable usage of leniolisib- and leniolisib-treated patients had lower odds of hypertension. rupress.org/jhi/article/...
October 26, 2026 at 5:30 PM
Santiago et al. utilize a large, limited, de-identified dataset to explore characteristics of patients with APDS. Hypertension was found in 28.5% of patients. There was variable usage of leniolisib- and leniolisib-treated patients had lower odds of hypertension. rupress.org/jhi/article/...
Pharming's leniolisib cut spleen volume by 26% across four genetic PID subtypes in Phase II, broadening its reach beyond the approved APDS label. Data presented at ESID 2026.
Pharming's leniolisib shows spleen and lung benefits in broader PIDs
Phase II topline data presented at ESID 2026 show leniolisib reduced spleen volume by 26% across four genetic PID subtypes beyond its approved APDS indication.
thebiotechtimes.com
October 22, 2026 at 2:46 PM
Pharming's leniolisib cut spleen volume by 26% across four genetic PID subtypes in Phase II, broadening its reach beyond the approved APDS label. Data presented at ESID 2026.
Open-label extension study of #leniolisib in activated PI3Kδ syndrome: Long-term efficacy and safety. New study from V. Koneti Rao, Gulbu Uzel @charlesuni.cuni.cz and colleagues:
rupress.org/jhi/article/...
#InbornErrorsOfImmunity
rupress.org/jhi/article/...
#InbornErrorsOfImmunity
October 21, 2026 at 1:45 PM
Open-label extension study of #leniolisib in activated PI3Kδ syndrome: Long-term efficacy and safety. New study from V. Koneti Rao, Gulbu Uzel @charlesuni.cuni.cz and colleagues:
rupress.org/jhi/article/...
#InbornErrorsOfImmunity
rupress.org/jhi/article/...
#InbornErrorsOfImmunity
Therapeutic progress !
Open-label extension study of leniolisib in activated PI3Kδ syndrome: Long-term efficacy and safety rupress.org/jhi/article/...
Open-label extension study of leniolisib in activated PI3Kδ syndrome: Long-term efficacy and safety rupress.org/jhi/article/...
Open-label extension study of leniolisib in activated PI3Kδ syndrome: Long-term efficacy and safety
Leniolisib, a PI3Kδ inhibitor, improves the pathophysiology of APDS. This publication demonstrates the safety and efficacy of leniolisib in patients with A
rupress.org
October 15, 2026 at 1:21 PM
Therapeutic progress !
Open-label extension study of leniolisib in activated PI3Kδ syndrome: Long-term efficacy and safety rupress.org/jhi/article/...
Open-label extension study of leniolisib in activated PI3Kδ syndrome: Long-term efficacy and safety rupress.org/jhi/article/...
#Leniolisib, a PI3Kδ inhibitor, improves the pathophysiology of APDS. Rao, Uzel @charlesuni.cuni.cz et al. demonstrate the safety and efficacy of leniolisib in patients with APDS ≥12 years of age receiving up to 7 years of treatment. rupress.org/jhi/article/...
#InbornErrorsOfImmunity
#InbornErrorsOfImmunity
October 14, 2026 at 7:00 PM
#Leniolisib, a PI3Kδ inhibitor, improves the pathophysiology of APDS. Rao, Uzel @charlesuni.cuni.cz et al. demonstrate the safety and efficacy of leniolisib in patients with APDS ≥12 years of age receiving up to 7 years of treatment. rupress.org/jhi/article/...
#InbornErrorsOfImmunity
#InbornErrorsOfImmunity
#Leniolisib, a PI3Kδ inhibitor, improves the pathophysiology of APDS. In @jhumimmunity.org, Rao, Uzel @charlesuni.cuni.cz et al. demonstrate the safety and efficacy of leniolisib in patients with APDS ≥12 years of age receiving up to 7 years of treatment. rupress.org/jhi/article/...
October 14, 2026 at 8:00 PM
#Leniolisib, a PI3Kδ inhibitor, improves the pathophysiology of APDS. In @jhumimmunity.org, Rao, Uzel @charlesuni.cuni.cz et al. demonstrate the safety and efficacy of leniolisib in patients with APDS ≥12 years of age receiving up to 7 years of treatment. rupress.org/jhi/article/...
#PHAR Pharming presents positive Phase II topline data for leniolisib in genetic PIDs with immune dysregulation at ESID 2026
https://www.stocktitan.net/news/PHAR/pharming-presents-positive-phase-ii-topline-data-for-leniolisib-in-lw83w8xsa72x.html?utm_source=stocktitan-bluesky&utm_medium=social
https://www.stocktitan.net/news/PHAR/pharming-presents-positive-phase-ii-topline-data-for-leniolisib-in-lw83w8xsa72x.html?utm_source=stocktitan-bluesky&utm_medium=social
Pharming presents positive Phase II topline data for leniolisib in genetic PIDs with immune dysregulation at ESID 2026
Stock Titan provides an ultra-fast stock market news feed and multiple tools for traders and investors.
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October 14, 2026 at 6:00 AM
#PHAR Pharming presents positive Phase II topline data for leniolisib in genetic PIDs with immune dysregulation at ESID 2026
https://www.stocktitan.net/news/PHAR/pharming-presents-positive-phase-ii-topline-data-for-leniolisib-in-lw83w8xsa72x.html?utm_source=stocktitan-bluesky&utm_medium=social
https://www.stocktitan.net/news/PHAR/pharming-presents-positive-phase-ii-topline-data-for-leniolisib-in-lw83w8xsa72x.html?utm_source=stocktitan-bluesky&utm_medium=social
Leniolisib and rapamycin in Activated PI3-Kinase-δ-syndrome: a retrospective ESID registry-based analysis. www.sciencedirect.com/science/arti... #hvhebron #upiip [Text complet]
www.sciencedirect.com
October 2, 2026 at 10:41 AM
Leniolisib and rapamycin in Activated PI3-Kinase-δ-syndrome: a retrospective ESID registry-based analysis. www.sciencedirect.com/science/arti... #hvhebron #upiip [Text complet]
FDA has accepted its supplemental New Drug Application (sNDA) for lower doses of Joenja® (leniolisib) to treat children aged 4 years and older...
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#FinTwit #FinancialDisclosure #6K
September 25, 2026 at 8:17 PM
FDA has accepted its supplemental New Drug Application (sNDA) for lower doses of Joenja® (leniolisib) to treat children aged 4 years and older...
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#FinTwit #FinancialDisclosure #6K
🟢 #$PHAR — Regulatory Designation
Priority Review Granted - Joenja Lower-Dose Pediatric sNDA
The FDA granted Priority Review to Pharming's sNDA for lower doses of Joenja (leniolisib) for children aged 4+ weighing 13-27 kg with APDS, alongside acceptance of the …
https://biostock.app/symbols/PHAR
Priority Review Granted - Joenja Lower-Dose Pediatric sNDA
The FDA granted Priority Review to Pharming's sNDA for lower doses of Joenja (leniolisib) for children aged 4+ weighing 13-27 kg with APDS, alongside acceptance of the …
https://biostock.app/symbols/PHAR
September 25, 2026 at 3:21 PM
🟢 #$PHAR — Regulatory Designation
Priority Review Granted - Joenja Lower-Dose Pediatric sNDA
The FDA granted Priority Review to Pharming's sNDA for lower doses of Joenja (leniolisib) for children aged 4+ weighing 13-27 kg with APDS, alongside acceptance of the …
https://biostock.app/symbols/PHAR
Priority Review Granted - Joenja Lower-Dose Pediatric sNDA
The FDA granted Priority Review to Pharming's sNDA for lower doses of Joenja (leniolisib) for children aged 4+ weighing 13-27 kg with APDS, alongside acceptance of the …
https://biostock.app/symbols/PHAR
▲ $PHAR Pharming Group: Positive Phase II data for leniolisib in PIDs showed a mean 26.4% spleen volume reduction. Data accepted as late-breaking abstract at ESID 2026, with a Phase II CVID readout expected in Q4 2026. [BULLISH]
September 23, 2026 at 1:17 AM
▲ $PHAR Pharming Group: Positive Phase II data for leniolisib in PIDs showed a mean 26.4% spleen volume reduction. Data accepted as late-breaking abstract at ESID 2026, with a Phase II CVID readout expected in Q4 2026. [BULLISH]
🚨The #FDA has approved #Joenja (leniolisib) as the first treatment for children aged 4-11 years with activated phosphoinositide 3-kinase delta syndrome (#APDS), a rare genetic disorder affecting the immune system.
📊Phase 3 study showed reduced lymphadenopathy and increased naïve B cells.
📊Phase 3 study showed reduced lymphadenopathy and increased naïve B cells.
September 14, 2026 at 9:00 AM
Pharming's leniolisib (Joenja) is now FDA-approved for children aged 4 to 11 with APDS, the first treatment cleared for this age group in the rare immunodeficiency.
Pharming wins FDA approval for Joenja in children aged 4 to 11 with APDS
The Netherlands-based biotech secured a supplemental approval making leniolisib the first FDA-approved treatment for younger children with the rare immunodeficiency APDS.
biotech.disruptsmedia.com
September 12, 2026 at 5:45 PM
Pharming's leniolisib (Joenja) is now FDA-approved for children aged 4 to 11 with APDS, the first treatment cleared for this age group in the rare immunodeficiency.
🟢 #$PHAR — Regulatory Approval
FDA Approval - Joenja for Pediatric APDS Ages 4-11 (40mg/50mg doses)
FDA approved Pharming's sNDA for 40 mg and 50 mg twice-daily dosing of Joenja (leniolisib) for children aged 4 to 11 years weighing at least 27 kg with APDS, mak…
https://biostock.app/symbols/PHAR
FDA Approval - Joenja for Pediatric APDS Ages 4-11 (40mg/50mg doses)
FDA approved Pharming's sNDA for 40 mg and 50 mg twice-daily dosing of Joenja (leniolisib) for children aged 4 to 11 years weighing at least 27 kg with APDS, mak…
https://biostock.app/symbols/PHAR
September 11, 2026 at 7:05 PM
🟢 #$PHAR — Regulatory Approval
FDA Approval - Joenja for Pediatric APDS Ages 4-11 (40mg/50mg doses)
FDA approved Pharming's sNDA for 40 mg and 50 mg twice-daily dosing of Joenja (leniolisib) for children aged 4 to 11 years weighing at least 27 kg with APDS, mak…
https://biostock.app/symbols/PHAR
FDA Approval - Joenja for Pediatric APDS Ages 4-11 (40mg/50mg doses)
FDA approved Pharming's sNDA for 40 mg and 50 mg twice-daily dosing of Joenja (leniolisib) for children aged 4 to 11 years weighing at least 27 kg with APDS, mak…
https://biostock.app/symbols/PHAR
This study used MCDA for Orphan Drugs to compare #leniolisib vs. standard of care in APDS. A panel found leniolisib superior in efficacy, safety, QoL, and cost, making it a valuable treatment.
journals.aboutscience.eu/index.php/gr...
journals.aboutscience.eu/index.php/gr...
January 29, 2025 at 10:16 AM
This study used MCDA for Orphan Drugs to compare #leniolisib vs. standard of care in APDS. A panel found leniolisib superior in efficacy, safety, QoL, and cost, making it a valuable treatment.
journals.aboutscience.eu/index.php/gr...
journals.aboutscience.eu/index.php/gr...
MHRA uses fast-track process to approve first medicine for rare immune disease APDS
www.pharmacy.biz/news/mhra-us...
www.pharmacy.biz/news/mhra-us...
MHRA Fast-Tracks Approval of Joenja for Rare Immune Disease APDS
Discover how the MHRA swiftly approved leniolisib phosphate (Joenja) to treat the rare immune disease APDS, bringing hope to UK patients.
www.pharmacy.biz
September 30, 2024 at 7:20 PM
MHRA uses fast-track process to approve first medicine for rare immune disease APDS
www.pharmacy.biz/news/mhra-us...
www.pharmacy.biz/news/mhra-us...
#$PHAR -17% [Pharming receives FDA Complete Response Letter for Joenja (leniolisib) in children aged 4-11 with APDS. Additional data needed on dosing and testing methods.] notreload.xyz/fda-requests...
FDA Requests More Data for Joenja in Kids with APDS
Pharming (PHAR) receives FDA Complete Response Letter for Joenja (leniolisib) in children aged 4-11 with APDS. Additional data needed on dosing and testing methods.
notreload.xyz
February 2, 2026 at 1:02 PM
#$PHAR -17% [Pharming receives FDA Complete Response Letter for Joenja (leniolisib) in children aged 4-11 with APDS. Additional data needed on dosing and testing methods.] notreload.xyz/fda-requests...
Will NHS England announce that at least 10 patients have started treatment with leniolisib for APDS by July 2, 2025? #18a39063bd27743d9d2b4fe3729f2190dc2ac45e2963960b13487beb7ac22c68
⇒Answer
⇒Answer
June 28, 2025 at 3:11 AM
Will NHS England announce that at least 10 patients have started treatment with leniolisib for APDS by July 2, 2025? #18a39063bd27743d9d2b4fe3729f2190dc2ac45e2963960b13487beb7ac22c68
⇒Answer
⇒Answer
Pharming onderuit op Damrak na tegenvaller rond geneesmiddel
Pharming onderuit op Damrak na tegenvaller rond geneesmiddel
AMSTERDAM (ANP) - Pharming ging vrijdag 7 procent onderuit op de Amsterdamse beurs. De biotechnoloog liet weten dat de Europese goedkeuring voor het middel leniolisib vertraging oploopt. Leniolisib is een behandeling voor APDS, een zeldzame aandoening aan het immuunsysteem. Volgens Pharming heeft het Europees Geneesmiddelenbureau (EMA) het positieve klinische voordeel en de veiligheid van leniolisib voor APDS-patiënten bevestigd, maar wil de toezichthouder meer informatie op het gebied van chemie, productie en controles.
nieuws.nl
May 31, 2024 at 8:04 AM
Pharming onderuit op Damrak na tegenvaller rond geneesmiddel
Clinical Experience with Use of the PI3Kdelta Inhibitor Leniolisib to Treat Immune Dysregulation in Patients with CVID and CVID-Like Disorders rupress.org/jhi/article/... #hvhebron #imm [Text complet]
Clinical Experience with Use of the PI3Kdelta Inhibitor Leniolisib to Treat Immune Dysregulation in Patients with CVID and CVID-Like Disorders
Objective. Approximately half of common variable immunodeficiency (CVID) patients develop debilitating and life-threatening clinical manifestations of immu
rupress.org
May 6, 2026 at 11:38 AM
Clinical Experience with Use of the PI3Kdelta Inhibitor Leniolisib to Treat Immune Dysregulation in Patients with CVID and CVID-Like Disorders rupress.org/jhi/article/... #hvhebron #imm [Text complet]
Value contribution of leniolisib in the Treatment of Activated PI3Kδ syndrome (APDS) in Spain using Multi-Criteria Decision Analysis (MCDA) scientiasalut.gencat.cat/handle/11351... #ScientiaVH
Value contribution of leniolisib in the Treatment of Activated PI3Kδ syndrome (APDS) in Spain using Multi-Criteria Decision Analysis (MCDA)
scientiasalut.gencat.cat
April 25, 2025 at 10:25 AM
Value contribution of leniolisib in the Treatment of Activated PI3Kδ syndrome (APDS) in Spain using Multi-Criteria Decision Analysis (MCDA) scientiasalut.gencat.cat/handle/11351... #ScientiaVH