U.S. flag An official website of the United States government

On Oct. 1, 2024, the FDA began implementing a reorganization impacting many parts of the agency. We are in the process of updating FDA.gov content to reflect these changes.

U.S. Food and Drug Administration
  1. Home
  2. Drugs
  3. Development & Approval Process | Drugs
  4. Drug Approvals and Databases
  5. Resources for Information | Approved Drugs
  6. FDA approves alpelisib for PIK3CA-related overgrowth spectrum
  1. Resources for Information | Approved Drugs

FDA approves alpelisib for PIK3CA-related overgrowth spectrum

 

On April 5, 2022, the Food and Drug Administration granted accelerated approval to alpelisib (Vijoice, Novartis Pharmaceuticals) for adult and pediatric patients two years of age and older with severe manifestations of PIK3CA-related overgrowth spectrum (PROS) who require systemic therapy.

Efficacy was evaluated using real-world data from EPIK-P1 (NCT04285723), a single-arm clinical study in patients two years of age and older with PROS who received alpelisib as part of an expanded access program for compassionate use. Eligible patients had clinical manifestations of PROS 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 a ≥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% (95% CI: 14, 44) had a radiological response at Week 24. Among responding patients, 60% had a response lasting 12 months or longer.  

The most common (≥ 10%) adverse reactions occurring in patients were diarrhea, stomatitis, and hyperglycemia.

The recommended alpelisib dosage for pediatric patients (2 to less than 18 years of age) is 50 mg taken orally once daily with food; for pediatric patients 6 years of age and older, the dose can be increased to 125 mg after 24 weeks if clinically indicated. The recommended dosage for adult patients (≥ 18 years) is 250 mg taken orally once daily with food.

View full prescribing information for Vijoice.

This review used the Real-Time Oncology Review (RTOR) 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.

This application was granted priority review, breakthrough designation and orphan drug designation. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.

For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov.

For information on the COVID-19 pandemic, see the following resources:

Follow the Oncology Center of Excellence on Twitter @FDAOncology.

Back to Top