First FDA approved treatment for PIK3CA-related overgrowth spectrum

This week, the U.S. Food and Drug Administration granted accelerated approval for Novartis’ PI3K inhibitor alpelisib for use in PIK3CA-related overgrowth spectrum (PROS). The approval covers the entire umbrella of PROS which includes M-CM / MCAP patients. This approval was based on results of data compiled from patients accessing the drug through compassionate use (also called managed access).

United States patients ages 2 and up will be able to now get a prescription for VIJOICE, the brand name for alpelisib for PROS.

Patients in other parts of the world may be able to access alpelisib through Novartis Managed Access, a request made by your doctor.

This is an extraordinary approval as it covers the treatment of an enormous range of symptoms across PROS conditions, and it was based on real-world evidence rather than a placebo controlled trial. There is still a lot that is unknown with regards treatment of specific symptoms. We hope that research continues to address many questions that will remain about treatment.

Relevant links:
FDA Announcement
Novartis Announcement
VIJOICE website with patient assistance program called VIP