Novartis announced that it received FDA approval for its Vijoice (alpelisib) PIK3CA-Related Overgrowth Spectrum (PROS) treatment.

Basel, Switzerland-based Novartis said in a news release that Vijoice is the first FDA-approved treatment for PROS, a spectrum of rare conditions characterized by overgrowth in blood vessel anomalies.

FDA approval followed real-world evidence from the EPIK-P1 retrospective chart review study demonstrating the success of Vijoice in reducing target lesion volume and the improvement in PROS-related symptoms and manifestations. At week 24, 27% of patients (10/37) achieved a confirmed a response to treatment, defined as 20% or greater reduction in the sum of PROS target lesion volume.

Nearly three-quarters of patients (74%) with imaging at baseline and week 24 showed some reduction in target lesion volume, while patients reported improvements in pain (90%), fatigue (76%), vascular malformation (79%), limb asymmetry (69%) and disseminated intravascular coagulation (55%).

The most common adverse events in the trial were diarrhea (16%), stomatitis (16%) and hyperglycemia (12%). The most common grade 3/4 adverse event as cellulitis (4%), with one case considered to be treatment-related.

“The approval of Vijoice marks a turning point for patients who, until now, have not had an approved therapy to specifically address their disease,” Novartis Innovative Medicines U.S. President Victor Bulto said in the release. “We are grateful to the physicians, patients and families who participated in the EPIK-P1 trial. We are continuing to invest in studies to advance the scientific understanding of PROS conditions and to understand the full potential of Vijoice.”