FDA Grants Accelerated Approval for Vijoice to Treat Rare Disorder

The FDA has granted accelerated approval to Novartis’ Vijoice (alpelisib) for the treatment of adult and pediatric patients 2 years of age and older with severe manifestations of PIK3CA-related overgrowth spectrum (PROS).

Vijoice, a kinase inhibitor, is the first FDA-approved treatment for PROS, a spectrum of rare conditions characterized by overgrowths and blood vessel anomalies impacting an estimated 14 people per million. The therapy works by inhibiting the PI3K pathway.

FDA approval was based on real-world evidence from EPIK-P1, a retrospective chart review study that showed 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. Nearly three in four patients with imaging at baseline and week 24 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, fatigue, vascular malformation, limb asymmetry, and disseminated intravascular coagulation. In EPIK-P1, the most common side effects were diarrhea, stomatitis, and hyperglycemia.

Novartis is conducting additional clinical trials. EPIK-P2 is a prospective phase 2 multi-center study with an upfront 16-week placebo-controlled period, and extension period to evaluate the safety, the efficacy and pharmacokinetics of Vijoice to treat pediatrics and adults with PROS. EPIK-P3 is a phase 2 study to assess long-term safety and efficacy of Vijoice in people with PROS who participated in EPIK-P1.

Novartis plans to offer a patient support program that includes assistance to access medication, financial resources for eligible patients and continued education.