Translational medicine. Hôpital Necker Enfants Malades/Université Paris Cité. US FDA and EMA approvals of alpelisib for PROS patients thanks to our works
Big news today: Vijoice (Alpelisib) has been approved for use by the EMA. This medication addresses an unmet medical need for patients with severe or life-threatening PIK3CA-related overgrowth spectrum (PROS) disorders
ema.europa.eu/en/news/first-…
First Drug for Severe Overgrowth Disorders Approved in Europe!
The European Medicines Agency has conditionally authorized the use of Vijoice (aka alpelisib/Novartis) for the treatment of patients with PROS disorders.
PROS = PIK3CA-related overgrowth spectrum. A diverse group of rare genetic conditions that are characterized by uncontrolled growth of some tissues.
They are caused by a mutation in the PIK3CA gene.
Vijoice is indicated for patients over two years of age with severe or life-threatening manifestations of PROS, requiring systemic therapy.
Vijoice showed a 20% tumor reduction in about 37.5% of patients.
This is a condition that has many unmet medical needs, and this is the first step in the right direction.
#PROS#GrowthDisorder#EMA#EuropeanMedicine
Following US FDA approval in April 2022, our group has reached another major milestone today with the EMA conditional approval of alpelisib for patients with severe and life-threatening PIK3CA-related overgrowth spectrum (PROS). Great news for EU patients
ema.europa.eu/en/news/first-…
Our latest paper just released. Congratulations to Ilaria, Charles and the entire team 🍾🥳🔥Oncogenic PIK3CA mutations shape an immunoregulatory microenvironment in mosaic overgrowth disorders academic.oup.com/pnasnexus/a…
What extraordinary news — I’m extremely proud and honored.
Thank you @eshgsociety for this very prestigious award, which I share with the entire team. 🎉🍾🥂
Aujourd’hui nous célébrions les 10 ans d’introduction de l’alpelisib (BYL719) chez Emmanuel, le premier patient PIK3CA traité dans le monde. Immense fierté🇫🇷
10 years on alpelisib for Emmanuel, the first ever PROS patient treated. Fantastic accomplishment for all of us 🎉🥳🍾🥂
Our latest article just released. Congratulations to @IlariaGalasso and @morin_gbrl 🍾🍾🎉🎊Vascular malformations: from genetics to therapeutics | EMBO Molecular Medicine embopress.org/doi/full/10.10…
Our latest review on kidney drug repositioning in @Kidney_Int. Congrats to the 2 outstanding contributors @morin_gbrl and @IsnardPierre1👏🍾🥂Drug repurposing in PIK3CA-related overgrowth spectrum: breakthroughs from overgrowth syndromes to kidney disease kidney-international.org/art…
#France2030 | Félicitations à Guillaume Canaud @CanaudLab, lauréat d’une Chaire d’excellence en Biologie/Santé pour son projet sur les malformations vasculaires liées à KRAS.
🎯Objectif : développer des thérapies ciblées pour une médecine ultra personnalisée. 👏🏼
📢#France2030 15 nouveaux chercheurs et chercheuses d'exception récompensés pour les « Chaires d’excellence en Biologie / Santé » !🧑🔬
Ce dispositif vise à renforcer l’attractivité de la recherche biomédicale française🧪🇫🇷🥼
Pour découvrir les lauréats👉swll.to/ilGDHI
Excellente nouvelle et merci pour votre soutien! Ce financement devrait accélérer l’identification de nouveaux traitements pour les malformations vasculaires secondaires a des mutations de KRAS 🍾🎊🥳
Wonderful news! This is a great funding for KRAS-vascular malformations! 🙏🙏
📢#France2030 15 nouveaux chercheurs et chercheuses d'exception récompensés pour les « Chaires d’excellence en Biologie / Santé » !🧑🔬
Ce dispositif vise à renforcer l’attractivité de la recherche biomédicale française🧪🇫🇷🥼
Pour découvrir les lauréats👉swll.to/ilGDHI
Here is our latest publication. Somatic PIK3R1 mutations in patients and treatment with alpelisib. Congratulations to the whole team and particularly to two very talented students @morin_gbrl and @Alexandre Garneau🥳🥂🍾
EMBO Molecular Medicine embopress.org/doi/full/10.10…
Key messages:
1-No fertility issues in 3 PROS patients with prolonged exposure to alpelisib
2-No embryotoxicity
3-Unfortunately, we observed disease recurrence following alpelisib withdrawal
4-But, no resistance to drug when alpelisib was reintroduced after pregnancies.
Happy to share our latest article:
« First report of successful pregnancies after treatment with alpelisib for PIK3CA-related overgrowth spectrum »
Congratulations to the whole team and particularly to @morin_gbrl 🥳🍾🎉
@ejhg_journalrdcu.be/epJZ1
Key messages 👇
Our latest paper is out today in @PNAS. Congratulations to Quitterie, Sophia, Marina, and the entire team!🍾🤩🥳PIK3CA gain-of-function mutation in Schwann cells leads to severe neuropathy and aerobic glycolysis through a non-cell autonomous effect | PNAS pnas.org/doi/10.1073/pnas.24…
[PUBLICATION] First report of successful pregnancies after treatment with alpelisib for PIK3CA-related overgrowth spectrum.
➡️Fertility preserved
➡️No embryotoxicity
➡️Disease remained responsive after treatment pause
📖Details here: nature.com/articles/s41431-0…