EMA backs plozasiran for rare lipid disorder with high pancreatitis risk
pharmafile | April 27, 2026 | News story | Research and Development |Â Â The European Medicines Agency (EMA), plozasiranÂ
The European Medicines Agency (EMA) has recommended granting a marketing authorisation for Redemplo (plozasiran) to treat adults with familial chylomicronaemia syndrome (FCS), a rare genetic condition linked to very high triglyceride levels and recurrent pancreatitis.
FCS prevents the body from breaking down fats, leaving patients at risk of severe abdominal pain and potentially life-threatening attacks of acute pancreatitis. The condition can also lead to complications including enlarged liver and spleen, diabetes, cognitive difficulties and skin deposits known as xanthomas.
Plozasiran is a small interfering RNA (siRNA) therapy designed to reduce production of APOC3, a protein that slows fat metabolism. By lowering APOC3 levels, the treatment aims to reduce triglycerides and, in turn, the risk of pancreatitis. It is administered as a subcutaneous injection every three months.
The EMA’s recommendation is based on a study involving 75 adults with FCS, all of whom were following a controlled diet. After 10 months, patients receiving plozasiran experienced an average reduction in triglyceride levels of 80%, compared with 17% in those given placebo. The effect was sustained over one year and up to at least 18 months.
The study also reported fewer cases of acute pancreatitis in patients treated with plozasiran compared with placebo, suggesting a potential clinical benefit beyond lipid lowering.
Unlike some existing treatments, plozasiran does not require genetic confirmation of FCS, which may broaden access for patients diagnosed based on clinical features.
The most commonly reported side effects included raised blood glucose levels, headache, nausea and injection-site reactions.
The recommendation from the EMA’s Committee for Medicinal Products for Human Use will now be reviewed by the European Commission, which will make a final decision on EU-wide approval. Access for patients will depend on national decisions on pricing and reimbursement.
