FDA D.I.S.C.O. Burst Edition: FDA approval of Vijoice (alpelisib) for adult and pediatric patients two years of age and older with severe manifestations of PIK3CA-related overgrowth spectrum who require systemic therapy
Welcome back to the D.I.S.C.O., FDA’s Drug Information Soundcast in Clinical Oncology, Burst Edition, brought to you by FDA’s Division of Drug Information in partnership with FDA’s Oncology Center of Excellence. Today we’ll provide another quick update on a recent FDA cancer drug approval.
On April 5, 2022, the FDA granted accelerated approval to alpelisib (brand name Vijoice) for adult and pediatric patients two years of age and older with severe manifestations of PIK3CA-related overgrowth spectrum who require systemic therapy.
Efficacy was evaluated using real-world data from EPIK-P1, a single-arm clinical study in patients two years of age and older with PIK3CA-related overgrowth spectrum who received alpelisib as part of an expanded access program for compassionate use. Eligible patients had clinical manifestations of PIK3CA-related overgrowth spectrum that were assessed by the treating physicians as severe or life-threatening and necessitating systemic treatment and had documented evidence of mutation in the PIK3CA gene. The efficacy of alpelisib was evaluated in a total of 37 patients with at least one target lesion identified on imaging performed within 24 weeks prior to receipt of the first dose.
The major efficacy outcome measure was the proportion of patients with radiological response at week 24 as determined by blinded independent central radiology review, defined as more than 20% reduction from baseline in the sum of measurable target lesion volume in up to 3 lesions confirmed by at least 1 subsequent imaging assessment. Duration of response was an additional efficacy outcome measure. Of the 37 patients included in the efficacy population, 27% had a radiological response at Week 24. Among responding patients, 60% had a response lasting 12 months or longer.
The most common adverse reactions reported in more than 10% of patients were diarrhea, stomatitis, and hyperglycemia.
This review used the Real-Time Oncology Review pilot program, which streamlined data submission prior to the filing of the entire clinical application, and the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.
Full prescribing information for these approvals can be found on the web at www.fda.gov/drugsatFDA.
Health care professionals should report serious adverse events to FDA’s MedWatch Reporting Program at www.fda.gov/medwatch.
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