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On April 5, 2022, the Food and Drug Administration (FDA) approved alpelisib (Vijoice, Novartis Pharmaceuticals) treatment for adult and pediatric patients aged two years and older with severe manifestations of the spectrum of PIK3CA-related overgrowth syndromes (PROS) requiring systemic treatment.

Efficacy was evaluated using a clinical trial of 37 patients over two years of age with clinical manifestations of PROS who received alpelisib. The patients’ symptoms were severe or life-threatening, required systemic treatment, and had documented evidence of mutation in the PIK3CA gene.

The primary measure of treatment efficacy was the proportion of patients with radiological response at week 24, defined as a ≥20% reduction from baseline in the sum of target lesion volume. Duration of response was an additional measure of efficacy. Of the 37 patients included in the efficacy population, 27% had a radiological response at week 24. Among patients who responded, 60% had a response that lasted 12 months or longer.

Treatment with alpelisib is presented as an effective treatment for patients aged 2 years and older with severe PROS manifestations requiring systemic treatment.


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