Novartis announces findings from a real-world study of alpelisib demonstrating clinical benefit in people with PIK3CA-Related Overgrowth Spectrum (PROS)

  • PROS is a spectrum of rare disorders caused by PIK3CA mutations and is characterized by atypical, visible overgrowths and anomalies in blood vessels, the lymphatic system and other tissues

  • At 24 weeks, 38% of patients achieved ≥20% reduction in the volume of the PROS lesions assessed in the primary endpoint analysis; no patients experienced disease progression or death
     
  • Alpelisib is the first potential treatment to specifically address the root cause of PROS conditions
  • EPIK-P1 study …
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