Translational medicine. Hôpital Necker Enfants Malades/Université Paris Cité. US FDA and EMA approvals of alpelisib for PROS patients thanks to our works

Paris, France
Joined June 2018
A huge thank you to the @eshgsociety for this extraordinary honor. I am deeply grateful and humbled to receive the Mendel Medal. This recognition is not mine alone, it belongs to my entire team, whose outstanding work, dedication, and passion made it possible. Thank you all.
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Guillaume Canaud (MD, PhD) retweeted
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-…
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Guillaume Canaud (MD, PhD) retweeted
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
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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-…
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What extraordinary news — I’m extremely proud and honored. Thank you @eshgsociety for this very prestigious award, which I share with the entire team. 🎉🍾🥂
The ESHG Award, ELPAG Award and the Mendel Lecturers of the #eshg2026 #hybridconference have talked to Mary Rice. Read the interviews and find out more about their backgrounds: 2026.eshg.org/speakers/speak…
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Bravo @casanova_lab So well deserved 🍾
We're proud to announce that infectious disease pioneer Jean-Laurent Casanova, MD, PhD, of @RockefellerUniv, @InstitutImagine, @hhmi_science, & @Casanova_Lab, is the distinguished recipient of the 2026 Mechthild Esser #NemmersPrize in Medical Science. 🔗spr.ly/6017hCLtR
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Merci
📢Journée mondiale des maladies rares | Focus sur le syndrome de Cloves avec @CanaudLab, docteur au sein de l’unité de Médecine translationnelle et thérapies ciblées @hopital_necker 👉 swll.to/u3rtlvF Cc @gouvernementFR @Sante_Gouv @SantePubliqueFr
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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 🎉🥳🍾🥂
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