A PIK3delta inhibitor for ALPS:
Leniolisib reduced lymphoproliferative disease in murine autoimmune lymphoproliferative syndrome url: rupress.org/jhi/article/...
Leniolisib reduced lymphoproliferative disease in murine autoimmune lymphoproliferative syndrome url: rupress.org/jhi/article/...
Leniolisib reduced lymphoproliferative disease in murine autoimmune lymphoproliferative syndrome | Journal of Human Immunity | Rockefeller University Press
Treating patients with ALPS can be challenging. This article examines the efficacy of leniolisib, a selective PI3Kδ inhibitor, in a murine model of ALPS, i
rupress.org
November 20, 2025 at 7:34 AM
A PIK3delta inhibitor for ALPS:
Leniolisib reduced lymphoproliferative disease in murine autoimmune lymphoproliferative syndrome url: rupress.org/jhi/article/...
Leniolisib reduced lymphoproliferative disease in murine autoimmune lymphoproliferative syndrome url: rupress.org/jhi/article/...
Expanding the scope of PI3K-δ inhibition: Leniolisib treatment in PRKCD deficiency. New report from Lodi, Guarnieri, Ricci, et al. at the University of Florence: rupress.org/jhi/article/...
April 28, 2026 at 6:44 PM
Expanding the scope of PI3K-δ inhibition: Leniolisib treatment in PRKCD deficiency. New report from Lodi, Guarnieri, Ricci, et al. at the University of Florence: rupress.org/jhi/article/...
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...
New hope for these patients:
Expanding the scope of PI3K-δ inhibition: Leniolisib treatment in PRKCD deficiency rupress.org/jhi/article/...
Expanding the scope of PI3K-δ inhibition: Leniolisib treatment in PRKCD deficiency rupress.org/jhi/article/...
Expanding the scope of PI3K-δ inhibition: Leniolisib treatment in PRKCD deficiency
Lodi et al. report leniolisib use, a PI3Kδ inhibitor approved for APDS, in a teenager with protein kinase C δ deficiency and progressive multiorgan immune
rupress.org
April 20, 2026 at 2:49 PM
New hope for these patients:
Expanding the scope of PI3K-δ inhibition: Leniolisib treatment in PRKCD deficiency rupress.org/jhi/article/...
Expanding the scope of PI3K-δ inhibition: Leniolisib treatment in PRKCD deficiency rupress.org/jhi/article/...
November A&R Clinical Connections
🍽️ Dietary Polyunsaturated Fatty Acid and the Risk of Rheumatoid Arthritis
💊 Precision Medicine in Pediatric Autoimmunity: Leniolisib Treatment of Childhood-Onset Lupus Nephritis Due to Activated PI3Kδ
https://acr.tw/4p4AkRZ
#Medsky
🍽️ Dietary Polyunsaturated Fatty Acid and the Risk of Rheumatoid Arthritis
💊 Precision Medicine in Pediatric Autoimmunity: Leniolisib Treatment of Childhood-Onset Lupus Nephritis Due to Activated PI3Kδ
https://acr.tw/4p4AkRZ
#Medsky
November 2, 2025 at 5:22 PM
November A&R Clinical Connections
🍽️ Dietary Polyunsaturated Fatty Acid and the Risk of Rheumatoid Arthritis
💊 Precision Medicine in Pediatric Autoimmunity: Leniolisib Treatment of Childhood-Onset Lupus Nephritis Due to Activated PI3Kδ
https://acr.tw/4p4AkRZ
#Medsky
🍽️ Dietary Polyunsaturated Fatty Acid and the Risk of Rheumatoid Arthritis
💊 Precision Medicine in Pediatric Autoimmunity: Leniolisib Treatment of Childhood-Onset Lupus Nephritis Due to Activated PI3Kδ
https://acr.tw/4p4AkRZ
#Medsky
Recently, leniolisib, a lifechanging new treatment for a rare immune disorder became available on the NHS.
We think its story is a great example of what curiosity-driven biomedical research can achieve when it's nurtured long term.
We think its story is a great example of what curiosity-driven biomedical research can achieve when it's nurtured long term.
Rare immune disorder treatment reaches NHS patients
A pioneering treatment for a rare immune disorder, developed through MRC-funded research, is now available on the NHS, transforming lives across UK and beyond.
www.ukri.org
August 29, 2025 at 8:46 AM
Recently, leniolisib, a lifechanging new treatment for a rare immune disorder became available on the NHS.
We think its story is a great example of what curiosity-driven biomedical research can achieve when it's nurtured long term.
We think its story is a great example of what curiosity-driven biomedical research can achieve when it's nurtured long term.
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]
Brief report demonstrates application of genetic testing and pathway-specific therapy to successfully treat a case of refractory class V lupus nephritis in an 18-year-old patient with activated PI3K-delta syndrome type 1
Arthritis & Rheumatology
doi.org/10.1002/art....
#Medsky #PrecisionMedicine
Arthritis & Rheumatology
doi.org/10.1002/art....
#Medsky #PrecisionMedicine
May 27, 2025 at 8:13 PM
Brief report demonstrates application of genetic testing and pathway-specific therapy to successfully treat a case of refractory class V lupus nephritis in an 18-year-old patient with activated PI3K-delta syndrome type 1
Arthritis & Rheumatology
doi.org/10.1002/art....
#Medsky #PrecisionMedicine
Arthritis & Rheumatology
doi.org/10.1002/art....
#Medsky #PrecisionMedicine
Lodi, Guarnieri, Ricci, et al from the University of Florence report the successful use of #leniolisib, a PI3Kδ inhibitor, to treat a teenager with protein kinase C δ deficiency & progressive multiorgan immune dysregulation, expanding on leniolisib’s potential indications rupress.org/jhi/article/...
April 21, 2026 at 4:43 PM
Lodi, Guarnieri, Ricci, et al from the University of Florence report the successful use of #leniolisib, a PI3Kδ inhibitor, to treat a teenager with protein kinase C δ deficiency & progressive multiorgan immune dysregulation, expanding on leniolisib’s potential indications rupress.org/jhi/article/...
Leniolisib reduced lymphoproliferative disease in murine autoimmune lymphoproliferative syndrome. New study from Chopie Hassan, Kevin S. Thorneloe (Pharming Group) and colleagues: rupress.org/jhi/article/...
#InbornErrorsOfImmunity
#InbornErrorsOfImmunity
November 24, 2025 at 3:15 PM
Leniolisib reduced lymphoproliferative disease in murine autoimmune lymphoproliferative syndrome. New study from Chopie Hassan, Kevin S. Thorneloe (Pharming Group) and colleagues: rupress.org/jhi/article/...
#InbornErrorsOfImmunity
#InbornErrorsOfImmunity
New study from Hassan et al. examines the efficacy of leniolisib, a selective PI3Kδ inhibitor, in a murine model of ALPS, illustrating a reduction in lymphoproliferation and disease-associated biomarkers such as elevated double-negative T cells. rupress.org/jhi/article/...
#InbornErrorsOfImmunity
#InbornErrorsOfImmunity
November 17, 2025 at 5:00 PM
New study from Hassan et al. examines the efficacy of leniolisib, a selective PI3Kδ inhibitor, in a murine model of ALPS, illustrating a reduction in lymphoproliferation and disease-associated biomarkers such as elevated double-negative T cells. rupress.org/jhi/article/...
#InbornErrorsOfImmunity
#InbornErrorsOfImmunity
Institute researchers, spanning research groups and facilities, contributed foundational knowledge about APDS, PI3-Kinase signalling and methodology central to the characterisation of APDS and the development of treatments.
www.babraham.ac.uk/news/2025/06...
www.babraham.ac.uk/news/2025/06...
June 27, 2025 at 11:23 AM
Institute researchers, spanning research groups and facilities, contributed foundational knowledge about APDS, PI3-Kinase signalling and methodology central to the characterisation of APDS and the development of treatments.
www.babraham.ac.uk/news/2025/06...
www.babraham.ac.uk/news/2025/06...
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...
First licensed treatment for ultra-rare immune disorder recommended
First licensed treatment for ultra-rare immune disorder recommended
NICE guidance published today recommends leniolisib (also called Joenja and made by Pharming) for treating activated phosphoinositide 3-kinase delta syndrome (APDS) in people 12 years and over.
www.nice.org.uk
March 13, 2025 at 3:00 PM
First licensed treatment for ultra-rare immune disorder recommended
#rarediseases #ultrararediseases #immunology #Joenja #leniolisib #PharmingTechnologies #raredisease #ActivatedPI3kinaseDeltaSyndrome #APDS #UniversityofCambridge #Addenbrookes #AnitaChandra #disregulatedimmunesystem #APDSpatients #SpecialisedCommissioning
www.bbc.co.uk/news/article...
www.bbc.co.uk/news/article...
New drug offers potential cure for ultra rare inherited condition
A teenage patient who helped discover a rare condition is the first to benefit from a new treatment.
www.bbc.co.uk
June 27, 2025 at 10:27 AM
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
🚀 Free alerts → https://gunpowderalerts.com/go?utm_source=bluesky&utm_medium=social&utm_campaign=high_priority_filing
#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...
🚀 Free alerts → https://gunpowderalerts.com/go?utm_source=bluesky&utm_medium=social&utm_campaign=high_priority_filing
#FinTwit #FinancialDisclosure #6K
🚀 Free alerts → https://gunpowderalerts.com/go?utm_source=bluesky&utm_medium=social&utm_campaign=high_priority_filing
#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
🟢 #$PHAR — Regulatory Approval
FDA Approval - Joenja for Pediatric APDS Ages 4-11 (40mg/50mg doses)
FDA approved Pharming's sNDA for 40 mg and 50 mg twice-daily dosing of Joenja (leniolisib) for children aged 4 to 11 years weighing at least 27 kg with APDS, mak…
https://biostock.app/symbols/PHAR
FDA Approval - Joenja for Pediatric APDS Ages 4-11 (40mg/50mg doses)
FDA approved Pharming's sNDA for 40 mg and 50 mg twice-daily dosing of Joenja (leniolisib) for children aged 4 to 11 years weighing at least 27 kg with APDS, mak…
https://biostock.app/symbols/PHAR
#$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...