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1. CN109562103 - BYL719 (ALPELISIB) FOR USE IN THE TREATMENT OF PIK3CA-RELATED OVERGROWTH SPECTRUM (PROS - CLOVES SYNDROME)

Office China
Application Number 201780011659.9
Application Date 17.02.2017
Publication Number 109562103
Publication Date 02.04.2019
Publication Kind A
IPC
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
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/-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
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
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
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
Applicants INSERM (INSTITUT NAT DE LA SANTE ET DE LA RECH MEDICALE)
国家医疗保健研究所
CENTRE NAT DE LA RECH SCIENT (CNRS)
国家科学研究中心
UNIVERSITE PARIS DESCARTES
雷卡尔巴黎大学
ASSIST PUBLIQUE-HOPITAUX DE PARIS (APHP)
巴黎公共救济院
Inventors CANAUD GUILLAUME
G·卡诺德
Agents 北京市金杜律师事务所 11256
北京市金杜律师事务所 11256
Priority Data 16305193.1 19.02.2016 EP
Title
(EN) BYL719 (ALPELISIB) FOR USE IN THE TREATMENT OF PIK3CA-RELATED OVERGROWTH SPECTRUM (PROS - CLOVES SYNDROME)
(ZH) 用于治疗PIK3CA相关过度生长综合征群的方法
Abstract
(EN)
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 methodof treating PROS in a subject in need thereof comprising the step of administrating the subject with a therapeutically effective amount of BYL719.

(ZH)
本发明涉及一种治疗PIK3CA相关过度生长综合征群(PROS),更具体的,先天性脂质性过度生长、血管畸形、表皮痣和脊柱/骨骼异常和/或脊柱侧凸(CLOVES)综合征的方法。目前,对于患者没有特异性的治疗,且没有PROS的动物模型以更好地了解该疾病的病理生理学。本发明人开发了PROS的遗传小鼠模型,其再现了人疾病并证明了BYL719的功效。基于这些结果,他们用BYL719治疗了患有严重CLOVES综合征的两名患者,一名成人和一名儿童。该药物在两名患者中对疾病具有强大的功效,诱导所有受影响器官的快速恢复。因此,本发明涉及一种治疗有需要的受试者的PROS的方法,包括向所述受试者施用治疗有效量的BYL719。