The European Medicines Agency (EMA) has taken a major step toward expanding treatment options for a rare and potentially life-threatening condition PROS.

EMA has recommended a conditional marketing authorisation in the EU for Vijoice, a medicine for PIK3CA-related overgrowth spectrum (PROS).

PROS is a group of rare genetic disorders marked by uncontrolled growth of body tissues, leading to malformations and lesions that can affect the skin, bones, blood vessels, and brain.

In its most severe forms, the condition can become extensive and life-threatening, impacting critical organs and vascular structures. At present, there is no approved medicine for PROS, with care largely limited to supportive measures such as surgery and procedures to manage abnormal blood vessels.

The disorder is driven by mutations in the PIK3CA gene, which trigger abnormal activity of the PI3 kinase enzyme involved in cell growth. This overactivity leads to excessive tissue growth and the formation of lesions and malformations.

Vijoice (alpelisib) targets this mechanism directly. It is a PI3 kinase inhibitor designed to block the overactive enzyme and reduce abnormal tissue growth. The treatment is available as tablets and granules, and is intended for patients with severe or life-threatening manifestations of PROS who require systemic therapy that acts throughout the body.

The EMA’s recommendation is based on evidence from a main study involving a retrospective review of medical records from 57 patients aged 2 years and older who received alpelisib through a compassionate use programme.

These patients had severe or life-threatening symptoms requiring systemic treatment. Effectiveness was measured by the proportion of patients showing at least a 20% reduction in the size of one to three target lesions after 24 weeks, primarily assessed through imaging scans. Among 32 patients evaluated at that point, 37.5% (12 out of 32) responded to treatment with Vijoice.

Further support for the medicine’s safety and effectiveness came from a follow-up study of patients enrolled in the compassionate use programme.

Side effects were consistent with the drug’s known safety profile, with the most commonly reported including hyperglycaemia, diarrhoea, headache, stomatitis, alopecia, dermatitis, dry skin, and nausea.

In reaching its conclusion, the EMA consulted clinical experts with experience treating PROS. The recommendation falls under the agency’s conditional marketing authorisation pathway, which allows earlier access to medicines addressing unmet medical needs while additional data are still being collected.

The company is expected to conduct further studies to confirm the long-term efficacy and safety of Vijoice in both adult and paediatric patients.

The Committee for Medicinal Products for Human Use (CHMP) has issued its opinion as an intermediary step, with the final decision now resting with the European Commission. If approved, individual EU member states will determine pricing and reimbursement based on national healthcare priorities.