MPR Pod out now! This week:
✅ Approvals for spinal muscular atrophy and CKD;
🧬 Joenja gains expanded approval;
💠 FDA to speed up LSD review for depression; and
🔵 Zasocitinib granted Priority Review. #Podcast
https://www.empr.com/news/mpr-weekly-dose-podcast-287/
✅ Approvals for spinal muscular atrophy and CKD;
🧬 Joenja gains expanded approval;
💠 FDA to speed up LSD review for depression; and
🔵 Zasocitinib granted Priority Review. #Podcast
https://www.empr.com/news/mpr-weekly-dose-podcast-287/
MPR Weekly Dose Podcast #287
Approvals for spinal muscular atrophy and chronic kidney disease; Joenja gains expanded approval in APDS; LSD derivative earns Breakthrough status to treat major depressive disorder; and zasocitinib i...
www.empr.com
September 18, 2026 at 9:28 PM
MPR Pod out now! This week:
✅ Approvals for spinal muscular atrophy and CKD;
🧬 Joenja gains expanded approval;
💠 FDA to speed up LSD review for depression; and
🔵 Zasocitinib granted Priority Review. #Podcast
https://www.empr.com/news/mpr-weekly-dose-podcast-287/
✅ Approvals for spinal muscular atrophy and CKD;
🧬 Joenja gains expanded approval;
💠 FDA to speed up LSD review for depression; and
🔵 Zasocitinib granted Priority Review. #Podcast
https://www.empr.com/news/mpr-weekly-dose-podcast-287/
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...
This week's MPR Weekly Dose Pod has got you covered:
✅ Approvals for spinal muscular atrophy and CKD;
🧬 Joenja gains expanded approval;
💠 FDA to speed up LSD review for depression; and
🔵 Zasocitinib granted Priority Review. #Podcast
https://www.empr.com/news/mpr-weekly-dose-podcast-287/
✅ Approvals for spinal muscular atrophy and CKD;
🧬 Joenja gains expanded approval;
💠 FDA to speed up LSD review for depression; and
🔵 Zasocitinib granted Priority Review. #Podcast
https://www.empr.com/news/mpr-weekly-dose-podcast-287/
MPR Weekly Dose Podcast #287
Approvals for spinal muscular atrophy and chronic kidney disease; Joenja gains expanded approval in APDS; LSD derivative earns Breakthrough status to treat major depressive disorder; and zasocitinib i...
www.empr.com
September 20, 2026 at 6:28 PM
This week's MPR Weekly Dose Pod has got you covered:
✅ Approvals for spinal muscular atrophy and CKD;
🧬 Joenja gains expanded approval;
💠 FDA to speed up LSD review for depression; and
🔵 Zasocitinib granted Priority Review. #Podcast
https://www.empr.com/news/mpr-weekly-dose-podcast-287/
✅ Approvals for spinal muscular atrophy and CKD;
🧬 Joenja gains expanded approval;
💠 FDA to speed up LSD review for depression; and
🔵 Zasocitinib granted Priority Review. #Podcast
https://www.empr.com/news/mpr-weekly-dose-podcast-287/
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...
#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
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
Leids Pharming mag tweede medicijn ook in EU op de markt brengen
Leids Pharming mag tweede medicijn ook in EU op de markt brengen
LEIDEN (ANP) - De Leidse biotechnoloog Pharming heeft toestemming gekregen om zijn tweede medicijn ook in de Europese Unie op de markt te brengen. Volgens het beursgenoteerde bedrijf is Joenja in het...
nieuws.nl
May 22, 2026 at 8:38 PM
Leids Pharming mag tweede medicijn ook in EU op de markt brengen
Pharming verlaagt omzetprognose door toegenomen concurrentie voor Ruconest
Hoewel in mindere mate dan in het eerste kwartaal, bleven de verkopen van het medicijn Ruconest in het tweede kwartaal onder druk staan door de fellere concurrentie. Pharmings ceo Fabrice Chouraqui heeft zich daarom...
Hoewel in mindere mate dan in het eerste kwartaal, bleven de verkopen van het medicijn Ruconest in het tweede kwartaal onder druk staan door de fellere concurrentie. Pharmings ceo Fabrice Chouraqui heeft zich daarom...
Pharming verlaagt omzetprognose door toegenomen concurrentie voor Ruconest
Dalende Ruconest-verkopen dwingen Pharming-ceo Fabrice Chouraqui tot lagere omzetprognose, terwijl Joenja sterk groeit. Lees hier de kerncijfers en vooruitzichten.
www.telegraaf.nl
July 30, 2026 at 7:04 AM
Pharming verlaagt omzetprognose door toegenomen concurrentie voor Ruconest
Hoewel in mindere mate dan in het eerste kwartaal, bleven de verkopen van het medicijn Ruconest in het tweede kwartaal onder druk staan door de fellere concurrentie. Pharmings ceo Fabrice Chouraqui heeft zich daarom...
Hoewel in mindere mate dan in het eerste kwartaal, bleven de verkopen van het medicijn Ruconest in het tweede kwartaal onder druk staan door de fellere concurrentie. Pharmings ceo Fabrice Chouraqui heeft zich daarom...
Excellent to be able to post some good news. This treatment for APDS sounds like it will lead the way for step changes in the treatment of rare & genetic diseases. news.sky.com/story/life-c...
'Life-changing' drug for teenager who lost mother to genetic condition
The new drug called Lleniolisib (Joenja) is a simple tablet taken twice a day and is the first ever targeted treatment for activated PI3-Kinase delta syndrome (APDS), a rare inherited disorder.
news.sky.com
June 27, 2025 at 8:04 AM
Excellent to be able to post some good news. This treatment for APDS sounds like it will lead the way for step changes in the treatment of rare & genetic diseases. news.sky.com/story/life-c...
🟢 #$PHAR — Regulatory Designation
Priority Review Granted - Joenja Lower-Dose Pediatric sNDA
The FDA granted Priority Review to Pharming's sNDA for lower doses of Joenja (leniolisib) for children aged 4+ weighing 13-27 kg with APDS, alongside acceptance of the …
https://biostock.app/symbols/PHAR
Priority Review Granted - Joenja Lower-Dose Pediatric sNDA
The FDA granted Priority Review to Pharming's sNDA for lower doses of Joenja (leniolisib) for children aged 4+ weighing 13-27 kg with APDS, alongside acceptance of the …
https://biostock.app/symbols/PHAR
September 25, 2026 at 3:21 PM
🟢 #$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
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
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
💊 #FDA Drug Approvals — September 2026
#Lirafugratinib, #Isembyld & #Zanvastro bring new approaches targeting FGFR, myostatin & RNA.
#Sevabertinib, #Camizestrant, #Imlunestrant, #Aqneursa & #Joenja expand therapeutic options across cancer, rare diseases & genetic disorders.
#Lirafugratinib, #Isembyld & #Zanvastro bring new approaches targeting FGFR, myostatin & RNA.
#Sevabertinib, #Camizestrant, #Imlunestrant, #Aqneursa & #Joenja expand therapeutic options across cancer, rare diseases & genetic disorders.
September 24, 2026 at 12:01 PM
💊 #FDA Drug Approvals — September 2026
#Lirafugratinib, #Isembyld & #Zanvastro bring new approaches targeting FGFR, myostatin & RNA.
#Sevabertinib, #Camizestrant, #Imlunestrant, #Aqneursa & #Joenja expand therapeutic options across cancer, rare diseases & genetic disorders.
#Lirafugratinib, #Isembyld & #Zanvastro bring new approaches targeting FGFR, myostatin & RNA.
#Sevabertinib, #Camizestrant, #Imlunestrant, #Aqneursa & #Joenja expand therapeutic options across cancer, rare diseases & genetic disorders.
#FDA approves #Joenja for children with activated phosphoinositide 3-kinase delta syndrome (APDS): is the first treatment approved for #APDS, a rare #primary #immunodeficiency. www.healio.com/news/...
September 15, 2026 at 11:00 AM
#FDA approves #Joenja for children with activated phosphoinositide 3-kinase delta syndrome (APDS): is the first treatment approved for #APDS, a rare #primary #immunodeficiency. www.healio.com/news/...
Pharming mag jonge kinderen met APDS in de VS behandelen
Toezichthouder FDA heeft de behandeling met Joenja voor kinderen van 4 tot 11 jaar met APDS in de VS goedgekeurd. Maker Pharming boekt in reactie hierop maandag een bescheiden koerswinst.
Toezichthouder FDA heeft de behandeling met Joenja voor kinderen van 4 tot 11 jaar met APDS in de VS goedgekeurd. Maker Pharming boekt in reactie hierop maandag een bescheiden koerswinst.
Pharming mag jonge kinderen met APDS in de VS behandelen
Toezichthouder FDA heeft de behandeling met Joenja voor kinderen van 4 tot 11 jaar met APDS in de VS goedgekeurd. Maker Pharming boekt in reactie hierop maandag een bescheiden koerswinst.
www.telegraaf.nl
September 14, 2026 at 8:44 AM
Pharming mag jonge kinderen met APDS in de VS behandelen
Toezichthouder FDA heeft de behandeling met Joenja voor kinderen van 4 tot 11 jaar met APDS in de VS goedgekeurd. Maker Pharming boekt in reactie hierop maandag een bescheiden koerswinst.
Toezichthouder FDA heeft de behandeling met Joenja voor kinderen van 4 tot 11 jaar met APDS in de VS goedgekeurd. Maker Pharming boekt in reactie hierop maandag een bescheiden koerswinst.
🚨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.
📰 #Finance
Pharming wins approval of Joenja for younger age group
seekingalpha.com
September 11, 2026 at 6:29 PM
📰 #Finance
The FDA has approved #$PHAR Joenja as the first U.S. treatment for children ages 4–11 with APDS who weigh at least 27 kg. notreload.xyz/xy/pharming-...
Pharming Joenja Approved for Kids 4–11 With APDS
The FDA has approved Pharming’s Joenja as the first U.S. treatment for children ages 4–11 with APDS who weigh at least 27 kg.
notreload.xyz
September 11, 2026 at 8:12 PM
The FDA has approved #$PHAR Joenja as the first U.S. treatment for children ages 4–11 with APDS who weigh at least 27 kg. notreload.xyz/xy/pharming-...
🟢 #$PHAR — Regulatory Approval
FDA Approval - Joenja for Pediatric APDS Ages 4-11 (40mg/50mg doses)
FDA approved Pharming's sNDA for 40 mg and 50 mg twice-daily dosing of Joenja (leniolisib) for children aged 4 to 11 years weighing at least 27 kg with APDS, mak…
https://biostock.app/symbols/PHAR
FDA Approval - Joenja for Pediatric APDS Ages 4-11 (40mg/50mg doses)
FDA approved Pharming's sNDA for 40 mg and 50 mg twice-daily dosing of Joenja (leniolisib) for children aged 4 to 11 years weighing at least 27 kg with APDS, mak…
https://biostock.app/symbols/PHAR
September 11, 2026 at 7:05 PM
🟢 #$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
Triodos Bank boekt fors meer winst en Pharming verovert de Japanse markt.
https://dgki.nl/posts/thursday/9165b3f-triodos-pharming/
https://dgki.nl/posts/thursday/9165b3f-triodos-pharming/
Triodos Bank boekt fors meer winst en Pharming verovert de Japanse markt.
Zeist, donderdag, 13 augustus 2026. Triodos Bank ziet de winst met 76 procent stijgen. Ondertussen mag Pharming zijn medicijn Joenja in Japan verkopen. Ook …
dgki.nl
August 13, 2026 at 10:28 AM
Triodos Bank boekt fors meer winst en Pharming verovert de Japanse markt.
https://dgki.nl/posts/thursday/9165b3f-triodos-pharming/
https://dgki.nl/posts/thursday/9165b3f-triodos-pharming/
Ceo Pharming: potentieel groter dan ooit ondanks omzetdomper
Ondanks de tegenvallende omzet in het tweede kwartaal en de hierdoor noodzakelijk geworden verlaging van de jaarprognose, kijkt ceo Fabrice Chouraqui van Pharming vol vertrouwen naar de toekomst. „Ik zie heel veel groeipotentieel en...
Ondanks de tegenvallende omzet in het tweede kwartaal en de hierdoor noodzakelijk geworden verlaging van de jaarprognose, kijkt ceo Fabrice Chouraqui van Pharming vol vertrouwen naar de toekomst. „Ik zie heel veel groeipotentieel en...
Ceo Pharming: potentieel groter dan ooit ondanks omzetdomper
Teleurstellende Ruconest-omzet drukt Pharming, maar Fabrice Chouraqui ziet miljardenkansen met Joenja en Napazimone. Lees hier zijn strategie.
www.telegraaf.nl
July 30, 2026 at 2:41 PM
Ceo Pharming: potentieel groter dan ooit ondanks omzetdomper
Ondanks de tegenvallende omzet in het tweede kwartaal en de hierdoor noodzakelijk geworden verlaging van de jaarprognose, kijkt ceo Fabrice Chouraqui van Pharming vol vertrouwen naar de toekomst. „Ik zie heel veel groeipotentieel en...
Ondanks de tegenvallende omzet in het tweede kwartaal en de hierdoor noodzakelijk geworden verlaging van de jaarprognose, kijkt ceo Fabrice Chouraqui van Pharming vol vertrouwen naar de toekomst. „Ik zie heel veel groeipotentieel en...
#$PHAR -17% [Pharming receives FDA Complete Response Letter for Joenja (leniolisib) in children aged 4-11 with APDS. Additional data needed on dosing and testing methods.] notreload.xyz/fda-requests...
FDA Requests More Data for Joenja in Kids with APDS
Pharming (PHAR) receives FDA Complete Response Letter for Joenja (leniolisib) in children aged 4-11 with APDS. Additional data needed on dosing and testing methods.
notreload.xyz
February 2, 2026 at 1:02 PM
#$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...