AU2024278598A1 — BYL719 (Alpelisib) for use in the treatment of PIK3CA-Related Overgrowth Spectrum (PROS - CLOVES syndrome)
Assigned to Centre National de la Recherche Scientifique CNRS · Expires 2025-01-16 · 1y expired
What this patent protects
OF THE INVENTION The present invention relates to a method of treating PIK3CA-Related Overgrowth 5 Spectrum (PROS) more particularly, Congenital, Lipomatous, Overgrowth, Vascular Malformations, Epidermal Nevi and Spinal/Skeletal Anomalies and/or Scoliosis (CLOVES) syndrome. T…
USPTO Abstract
OF THE INVENTION The present invention relates to a method of treating PIK3CA-Related Overgrowth 5 Spectrum (PROS) more particularly, Congenital, Lipomatous, Overgrowth, Vascular Malformations, Epidermal Nevi and Spinal/Skeletal Anomalies and/or Scoliosis (CLOVES) 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 10 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 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. 15
Drugs covered by this patent
- Piqray (alpelisib) · Novartis
Bibliographic data sourced from FDA Orange Book + USPTO public records. Plain-English summary generated by AI grounded in source text. Patent term extensions (PTR, SPC, pediatric) may shift the effective expiry. Not legal advice.
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