The European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use has recommended a conditional marketing authorization for Vijoice (alpelisib, Novartis) for the treatment of patients with severe PIK3CA-related overgrowth spectrum (PROS) disorders.
Vijoice is indicated for the treatment of adults and children aged 2 years and older with severe or life-threatening manifestations of PROS who require systemic therapy.
PROS is a diverse group of rare genetic conditions that are characterized by uncontrolled growth of some tissues in the body. PROS is caused by mutations in the PIK3CA gene, which lead to the production of an abnormal form of an enzyme (phosphatidylinositol-3-kinase [PI3K]) that is involved in cell growth. This abnormal form of PI3K causes the overgrowth of lesions and malformations that can affect the skin, bones, blood vessels, and brain. The severity of PROS ranges from localized overgrowth to severe, extensive, and life‑threatening overgrowth affecting important organs or blood vessels.
There is currently no authorized medicine for PROS and treatment consists of supportive care, which includes surgery and procedures to block overgrown blood vessels.
Reduces Abnormal Tissue Growth
The active substance in Vijoice is alpelisib, an antineoplastic agent, PI3K inhibitor. Alpelisib blocks the activity of the PI3K enzyme, thereby reducing the abnormal growth of tissues.
In the EU, alpelisib is already authorized as Piqray (Novartis) for the treatment of certain forms of breast cancer with a PIK3CA mutation.
For the treatment of PROS, Vijoice will be available as 50 mg, 125 mg, and 200 mg film-coated tablets, and 50 mg granules.
Its most common side effects include hyperglycemia, diarrhea, headache, stomatitis, alopecia, dermatitis, dry skin, and nausea.
Positive Treatment Response
The benefits of Vijoice are based on the results of the EPIK-P1 study. This was a retrospective review of medical records of patients aged 2 years and older with severe or life-threatening symptoms of PROS that required systemic treatment and received alpelisib through a compassionate use program.
The main measure of effectiveness was the number of patients who had at least a 20% reduction in the size of 1-3 abnormal tumors after 24 weeks of treatment. After this time, 37.5% of patients had experienced a ≥ 20% reduction in target lesion volume. Additional clinical benefit independent from lesion volume reduction was observed across the full study population.
A conditional marketing authorization is one of the EU regulatory mechanisms to facilitate early access to medicines that fulfil an unmet medical need. It allows the recommendation of a marketing authorization with less complete data than is normally expected, if the benefit of a medicine’s immediate availability to patients outweighs the risk inherent in the fact that not all the data are yet available.
The EMA emphasized that the company will carry out a study to further confirm the efficacy and safety of Vijoice in the treatment of adult and pediatric patients with PROS.
Rob Hicks is a retired National Health Service doctor. A well-known TV and radio broadcaster, he has written several books and has regularly contributed to national newspapers, magazines, and online publications. He is based in the United Kingdom.
