NVS: Study On Alpelisib Shows Clinical Benefit In People With PIK3CA-Related Overgrowth Spectrum

(RTTNews) - Novartis (NVS) announced findings from a real-world study, which evaluated the safety and efficacy of alpelisib for people living with PIK3CA-Related Overgrowth Spectrum or PROS who received treatment daily for at least 24 weeks.

Results from EPIK-P1 showed alpelisib effectively reduced volume of clinically significant PROS-related lesions and improved signs and symptoms in pediatric and adult patients.

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.

EPIK-P1 is a global, site-based retrospective non-interventional medical chart review of pediatric and adult male and female patients aged 2 years or older with PIK3CA-Related Overgrowth Spectrum who received alpelisib via a compassionate use program. The primary endpoint analysis conducted at week 24 in patients with complete cases showed 38% of patients achieving a response to treatment which was defined as 20% or greater reduction in the sum of PROS target lesion volume. Nearly three in four patients or 74% showed some reduction in target lesion volume, with a mean reduction of 13.7%, and no patients experienced disease progression at time of primary analysis.

The U.S. Food and Drug Administration granted alpelisib Breakthrough Therapy Designation on November 13, 2019, and discussions with FDA about alpelisib for PROS are ongoing.