Guillaume Canaud (MD, PhD)

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

[Guillaume Canaud (MD, PhD) @CanaudLab]

@NeilVasan Thank you Neil! I will be in NYC (Columbia) next September we can try to catch up

[Guillaume Canaud (MD, PhD) @CanaudLab]

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
https://t.co/g7zzBe0sen

[GoPI3Ks (Genetic Overgrowth PI3K Support) @GoPI3Ks]

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

https://t.co/HEMPgL6TiF

[Public Meducation @PubMeducation]

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

[Guillaume Canaud (MD, PhD) @CanaudLab]

@rkramann Thank you Rafael!

[Guillaume Canaud (MD, PhD) @CanaudLab]

@SanfordMSimon Thank you Simon! I Hope to catch you up soon
This was a long road

[Guillaume Canaud (MD, PhD) @CanaudLab]

@rkramann Thank you Rafael!

[Guillaume Canaud (MD, PhD) @CanaudLab]

Our latest paper just released. Congratulations to Ilaria, Charles and the entire team 🍾🥳🔥Oncogenic PIK3CA mutations shape an immunoregulatory microenvironment in mosaic overgrowth disorders https://t.co/F9Ykt7Yrr7

[Guillaume Canaud (MD, PhD) @CanaudLab]

@SanfordMSimon Thank you so much Sandy! I hope you’re doing well

[Guillaume Canaud (MD, PhD) @CanaudLab]

What extraordinary news — I’m extremely proud and honored.
Thank you @eshgsociety for this very prestigious award, which I share with the entire team. 🎉🍾🥂

[European Society of Human Genetics (ESHG) @eshgsociety]

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: https://t.co/6YZuTjpoTQ

[Guillaume Canaud (MD, PhD) @CanaudLab]

Bravo @casanova_lab
So well deserved 🍾

[Northwestern Feinberg School of Medicine @NUFeinbergMed]

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.

🔗https://t.co/zERwIaxdjx

[Guillaume Canaud (MD, PhD) @CanaudLab]

Merci

[Ministère Enseignement supérieur/Recherche/Espace @sup_recherche]

📢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

👉 https://t.co/xWhb6ImGuz

Cc @gouvernementFR @Sante_Gouv @SantePubliqueFr

[Guillaume Canaud (MD, PhD) @CanaudLab]

@ruth_hook_ Thank you!

[Guillaume Canaud (MD, PhD) @CanaudLab]

@NeilVasan 🙏

[Guillaume Canaud (MD, PhD) @CanaudLab]

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 🎉🥳🍾🥂

[Guillaume Canaud (MD, PhD) @CanaudLab]

@NeilVasan @Shalinda_Fdo @SABCSSanAntonio @Perlmutter_CC @PIK3CApathbreak Congratulations Neil and team. This is very interesting and consistent with our observation in PIK3R1 related overgrowth syndromes

https://t.co/IuLnb9wbB6

[Guillaume Canaud (MD, PhD) @CanaudLab]

@rkramann @ERAkidney Thank you so much Rafael 😊 It is always a pleasure to see you, even though it was too short. Cheers

[Guillaume Canaud (MD, PhD) @CanaudLab]

Our latest article just released. Congratulations to @IlariaGalasso and @morin_gbrl 🍾🍾🎉🎊Vascular malformations: from genetics to therapeutics | EMBO Molecular Medicine https://t.co/GoYB05pWxR

[Guillaume Canaud (MD, PhD) @CanaudLab]

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 https://t.co/vvNXvluiVo

[Guillaume Canaud (MD, PhD) @CanaudLab]

Wonderful team🙏🍾❤️