FDA Approves Alpelisib as First, Only Treatment for Select Patients with PIK3CA-Related Overgrowth Spectrum

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

Alpelisib is the first FDA-approved treatment for PROS, which is a range of rare conditions characterized by overgrowths and blood vessel anomalies impacting approximately 14 people per million, according to Novartis. Under the guidelines of the accelerated approval program, continued approval may be dependent upon verification and description of clinical benefit from confirmatory evidence.

PROS management may affect quality of life and can be challenging to manage because it requires a multidisciplinary team and prior interventions were more focused on symptom management for patients.

“Today’s approval of the first treatment for PROS offers hope for a better quality of life to patients and families affected by these rare conditions,” said Kristen Davis, executive director of CLOVES Syndrome Community, in a press release. “PROS conditions can be debilitating and disabling and can result in disruptions to everyday activities. Until today, often the only treatment options for patients were surgical or interventional radiology procedures.”

The approval was based on data from EPIK-P1, a chart review study that showed patients treated with alpelisib 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 achieved a confirmed response to treatment, or 20% or greater reduction in the sum of PROS target lesion volume. In addition, at week 24, the researchers observed patient improvements in pain, fatigue, vascular malformation, limb asymmetry, and disseminated intravascular coagulation.

The most common adverse events (AEs) in EPIK-P1 of any grade were diarrhea, stomatitis, and hyperglycemia, and the most common grade 3/4 AE was cellulitis.

REFERENCE

FDA approves Novartis Vijoice® (alpelisib) as first and only treatment for select patients with PIK3CA-Related Overgrowth Spectrum (PROS). Novartis. April 6, 2022. Accessed April 6, 2022. https://www.novartis.com/news/media-releases/fda-approves-novartis-vijoice-alpelisib-first-and-only-treatment-select-patients-pik3ca-related-overgrowth-spectrum-pros