The US Food and Drug Administration has given an accelerated approval to Novartis for a new drug to treat a rare group of genetic diseases known as PIK3CA-related overgrowth spectrum (PROS). The drug, Vijoice (alpelisib), was approved on the basis of real-world evidence from patients who received alpelisib as part of a compassionate use programme.
The PROS group of disorders cause overgrowth in parts of the body due to mutations in the PIK3CA gene. Vijoice is a kinase inhibitor designed to block the PI3K pathway and predominantly the P13K-alpha isoform.