General Information

Vijoice (alpelisib) is a kinase inhibitor.

Vijoice is specifically indicated for the treatment of adult and pediatric patients 2 years of age and older with severe manifestations of PIK3CARelated Overgrowth Spectrum (PROS) who require systemic therapy.

Vijoice is supplied as tablets for oral administration. The recommended dose for pediatric patients (2 to less than 18 years of age) is 50 mg taken orally once daily with food. The recommended dose for adult patients: 250 mg taken orally once daily with food.

Mechanism of Action

Vijoice (alpelisib) is an inhibitor of phosphatidylinositol-3-kinase (PI3K) with inhibitory activity predominantly against PI3Kα. Gain-of-function mutations in the gene encoding the catalytic α-subunit of PI3K (PIK3CA) lead to activation of PI3Kα and Akt-signaling, cellular transformation and the generation of tumors in in vitro and in vivo models. Activating mutations in PIK3CA have been found to induce a spectrum of overgrowths and malformations comprising a wide group of clinically recognizable disorders commonly known as PROS.

Clinical Trial Results

The FDA granted accelerated approval to Vijoice. The FDA’s accelerated approval was based on real-world evidence from EPIK-P1, https://clinicaltrials.gov/ct2/show/NCT04285723 a retrospective chart review study. Patients treated with Vijoice experienced reduced target lesion volume and improvement in PROS-related symptoms and manifestations. The primary endpoint analysis conducted at week 24 showed 27% of patients (10/37) achieved a confirmed response to treatment, defined as 20% or greater reduction in the sum of PROS target lesion volume. Nearly three in four patients with imaging at baseline and week 24 (74%, 23/31) 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. Additionally, at week 24, investigators observed patient improvements in pain (90%, 20/22), fatigue (76%, 32/42), vascular malformation (79%, 30/38), limb asymmetry (69%, 20/29), and disseminated intravascular coagulation (55%, 16/29). These improvements were observed in subsets of patients across the study population (n=57) who reported symptoms at baseline and at week 24.