First medicine to treat rare uncontrolled growth of body tissues

22 May 2026

Vijoice addresses an unmet medical need for patients with severe or life-threatening PIK3CA-related overgrowth spectrum (PROS) disorders

EMA has recommended granting a conditional marketing authorisation in the European Union (EU) for Vijoice to treat adults and children aged 2 years and older with severe or life-threatening PIK3CA-related overgrowth spectrum (PROS) disorders.

PROS is a diverse group of rare genetic conditions that are characterised by uncontrolled growth of some tissues in the body, causing malformations and lesions affecting the skin, bones, blood vessels and brain. The severity of PROS ranges from localised overgrowth to severe, extensive and life‑threatening overgrowth affecting important organs or blood vessels.

There is currently no authorised medicine for PROS and treatment consists of supportive care, including surgery and procedures to block overgrown blood vessels.

PROS is caused by mutations in a gene called PIK3CA, which lead to the production of an abnormal form of an enzyme (PI3 kinase) that is involved in cell growth. This abnormal form of PI3 kinase causes the overgrowth of tissues (lesions) and malformations.

The active substance in Vijoice, alpelisib, is a PI3 kinase inhibitor that works by blocking the activity of the enzyme, thereby reducing the abnormal growth of tissues.

Vijoice is available as tablets and granules and is for use in people with severe or life‑threatening manifestations of PROS who require systemic therapy (therapy that affects the whole body).

EMA’s recommendation is based on the results of a main study which involved the retrospective review of medical records from 57 patients aged 2 years and older who received alpelisib as part of a compassionate use programme. The patients had severe or life-threatening symptoms of PROS that required systemic treatment. The main measure of effectiveness was the number of patients who, after 24 weeks of treatment, had a reduction of at least 20% in the size of 1 to 3 abnormal growths or lesions, based mainly on imaging scans. Of the 32 patients who had an assessment at 24 weeks, 37.5% (12 out of 32) had responded to treatment with Vijoice.

A follow-up study involving patients who had taken part in the compassionate use programme provided further supportive evidence about the medicine’s safety and efficacy.

The most common side effects reported with Vijoice were hyperglycaemia (high blood sugar levels), diarrhoea, headache, stomatitis (inflammation of the lining of the mouth), alopecia (hair loss), dermatitis (inflammation of the skin), dry skin and nausea.

In reaching its decision, EMA consulted clinical experts with experience in treating PROS.

Vijoice is recommended for a conditional marketing authorisation, one of the EU regulatory mechanisms to facilitate early access to medicines that fulfil an unmet medical need. This type of approval allows the Agency to recommend a medicine for marketing authorisation with less complete data than 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 company will carry out a study to further confirm the efficacy and safety of Vijoice in the treatment of adult and paediatric patients with PROS.

The opinion adopted by the CHMP is an intermediary step on Vijoice’s path to patient access. The opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role or use of this medicine in the context of the national health system of that country.

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Notes

• The applicant for Vijoice is Novartis Europharm Limited.
• Vijoice was designated as an orphan medicinal product for PROS on 26 March 2021.
• Following this positive CHMP opinion, the Committee for Orphan Medicinal Products (COMP) will assess whether the orphan designation should be maintained.
• In the EU, alpelisib is already authorised as Piqray for the treatment of certain forms of breast cancer with a PIK3CA mutation, together with another medicine called fulvestrant.