EMA Recommends Redemplo (Plozasiran) for Adults with FCS

New medicine to reduce triglycerides in adults with familial chylomicronaemia syndrome

24 April 2026

Redemplo provides new treatment option for patients with a high unmet medical need News Human Medicines

EMA has recommended granting a marketing authorisation in the European Union (EU) for Redemplo (plozasiran) to treat adults with familial chylomicronaemia syndrome (FCS).

FCS is a rare inherited disease that prevents the body from breaking down lipids (fats). People with this condition have extremely high levels of triglycerides in their blood. This causes a range of symptoms, including severe abdominal pain, potentially fatal attacks of acute pancreatitis, hepatosplenomegaly (enlargement of liver and spleen), diabetes, lack of concentration, memory loss and fat-filled spots on the skin (called xanthomas).

People with FCS must strictly limit their fat intake through diet. However, this is not always feasible nor sufficiently effective to reduce the level of triglycerides and prevent pancreatitis. Traditional lipid-lowering medications have minimal impact when it comes to reducing triglyceride levels in patients with FCS.

The active substance in Redemplo, plozasiran, is a first-in-class small interfering RNA (siRNA) designed to block the production of APOC3, a protein that slows down the breakdown of fats. By blocking the production of this protein, Redemplo reduces the level of triglycerides in the blood and, as a result, fat accumulation in the body, which is expected to reduce the risk of pancreatitis. Redemplo is given every three months as a subcutaneous injection.

Although other authorised medicines can help people with FCS confirmed by genetic testing, Redemplo does not require genetic confirmation of the condition, thus providing a treatment option for more adults with FCS and addressing the unmet medical need in these patients.

EMA’s recommendation is based on data from one main study involving 75 adults with FCS. All patients in the study were on a controlled diet in addition to receiving Redemplo or placebo (a dummy treatment). After 10 months of treatment, patients receiving Redemplo had an average reduction in the blood level of triglycerides of 80%, compared with an average reduction of 17% in patients given placebo. This effect, which was observed in both genetically confirmed FCS and FCS confirmed by clinical diagnosis of signs and symptoms, was maintained during the one-year duration of the study and up to at least 18 months. The study also showed that there were significantly fewer cases of acute pancreatitis in patients using Redemplo compared with those using placebo.

The most common side effects reported with Redemplo were hyperglycaemia (high blood sugar levels), headache, nausea and injection-site reaction.

The opinion adopted by EMA’s human medicines committee (CHMP) is an intermediary step on Redemplo’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.

Notes

• The applicant for Redemplo is Arrowhead Pharmaceuticals Ireland Limited.
• Redemplo was designated as an orphan medicinal product for the treatment of familial chylomicronaemia syndrome on 19 July 2021.
• Following this positive CHMP opinion, the Committee for Orphan Medicinal Products (COMP) will assess whether the orphan designation should be maintained.

News

• Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 20-23 April 2026

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