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1. WO2017140828 - BYL719 (ALPELISIB) FOR USE IN THE TREATMENT OF PIK3CA-RELATED OVERGROWTH SPECTRUM (PROS - CLOVES SYNDROME)

Publication Number WO/2017/140828
Publication Date 24.08.2017
International Application No. PCT/EP2017/053587
International Filing Date 17.02.2017
IPC
A61K 31/44 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
435having six-membered rings with one nitrogen as the only ring hetero atom
44Non-condensed pyridines; Hydrogenated derivatives thereof
A61P 43/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
43Drugs for specific purposes, not provided for in groups A61P1/-A61P41/108
CPC
A61K 31/44
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
435having six-membered rings with one nitrogen as the only ring hetero atom
44Non condensed pyridines; Hydrogenated derivatives thereof
A61K 31/4439
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
435having six-membered rings with one nitrogen as the only ring hetero atom
44Non condensed pyridines; Hydrogenated derivatives thereof
4427containing further heterocyclic ring systems
4439containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
A61P 19/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
19Drugs for skeletal disorders
A61P 35/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
35Antineoplastic agents
A61P 43/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
43Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
A61P 9/14
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
9Drugs for disorders of the cardiovascular system
14Vasoprotectives; Antihaemorrhoidals; Drugs for varicose therapy; Capillary stabilisers
Applicants
  • INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) [FR]/[FR]
  • CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS) [FR]/[FR]
  • UNIVERSITÉ PARIS DESCARTES [FR]/[FR]
  • ASSISTANCE PUBLIQUE-HÔPITAUX DE PARIS (APHP) [FR]/[FR]
Inventors
  • CANAUD, Guillaume
Agents
  • COLLIN, Matthieu
Priority Data
16305193.119.02.2016EP
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) BYL719 (ALPELISIB) FOR USE IN THE TREATMENT OF PIK3CA-RELATED OVERGROWTH SPECTRUM (PROS - CLOVES SYNDROME)
(FR) BYL719 (ALPELISIB) POUR UNE UTILISATION DANS LE TRAITEMENT DES MALADIES DE LA FAMILLE DES HYPERTROPHIES LIÉES À PIK3CA (PROS - SYNDROME DE CLOVES)
Abstract
(EN)
- 20 - ABSTRACT OF THE INVENTION METHODS FOR THE TREATMENT OF PIK3CA-RELATED 5 OVERGROWTH SPECTRUM The present invention relates to a method of treating PIK3CA-Related Overgrowth Spectrum (PROS) more particularly, Congenital, Lipomatous, Overgrowth, Vascular Malformations, Epidermal Nevi and Spinal/Skeletal Anomalies and/or Scoliosis (CLOVES) 10 syndrome. To date, there are no specific treatments for patients and no animal models of PROS to better understand the physiopathology of the disorder. Inventors developed a genetic mouse model of PROS that recapitulates the human disease and demonstrated the efficacy of BYL719. Based on these results they treated two patients, one adult and one child, with severe CLOVES syndrome using BYL719. The drug had a robust efficiency on disease in the 15 two patients inducing quick recovery of all affected organs. Thus, the invention relates to a method of treating PROS in a subject in need thereof comprising the step of administrating the subject with a therapeutically effective amount of BYL719.
(FR)
- 20 - ABRÉGÉ DES MÉTHODES DE L'INVENTION POUR LE TRAITEMENT DES MALADIES DE LA FAMILLE DES HYPERTROPHIES LIÉES À PIK3CA La présente invention concerne une méthode de traitement des maladies de la famille des hypertrophies liées à PIK3CA (PROS), plus particulièrement le syndrome d'excroissance lipomateuse congénitale-malformation vasculaire-naevus épidermique-anomalie rachidienne (CLOVES) 10. À ce jour, il n'existe pas de traitement spécifique pour les patients ni de modèles animaux de PROS qui puisse permettre de mieux comprendre la physiopathologie de cette pathologie. Les inventeurs ont développé par génie génétique un modèle murin de PROS qui reproduit la maladie humaine et ont démontré l'efficacité de BYL719. Sur la base de ces résultats ils ont traité deux patients, un adulte et un enfant, atteint d'un syndrome de CLOVES sévère, à l'aide de BYL719. Le médicament a montré une efficacité robuste sur la maladie chez les deux patients, induisant une récupération rapide de tous les organes affectés. Ainsi, l'invention concerne une méthode permettant de traiter les PROS chez le patient le nécessitant, comprenant l'étape d'administration au patient d'une quantité thérapeutiquement efficace de BYL719.
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