The European Medicines Agency recommends a new drug for adults with familial chylomicronemia syndrome.

The European Medicines Agency has recommended the authorization in the European Union of Redemplo, a new medication for adults with familial chylomicronemia syndrome, a rare hereditary disease that can cause potentially fatal acute pancreatitis.

In short

EMA recommends granting a marketing authorization in the EU for Redemplo, intended for adults with familial chylomicronemia syndrome.

The disease prevents the body from breaking down lipids and leads to extremely high levels of triglycerides in the blood.

Redemplo contains plozasiran, a first-in-class siRNA treatment that blocks the production of the APOC3 protein.

In the main study, patients treated with Redemplo had an average reduction of 80% in triglycerides after 10 months, compared to 17% in the placebo group.

The CHMP opinion will be sent to the European Commission, which will decide on the marketing authorization at the EU level.

EMA announced on April 24, 2026, that it recommended the authorization of Redemplo for the treatment of adults with familial chylomicronemia syndrome, also known by the acronym FCS. The recommendation was adopted by the EMA's Committee for Medicinal Products for Human Use (CHMP) and represents an intermediate step before a final decision by the European Commission.

Familial chylomicronemia syndrome is a rare hereditary disease that prevents the body from breaking down lipids. Affected individuals have extremely high levels of triglycerides in the blood, which can cause severe abdominal pain, episodes of potentially fatal acute pancreatitis, hepatosplenomegaly, diabetes, lack of concentration, memory loss, and xanthomas, which are skin lesions filled with fat.

Patients with FCS must strictly limit their fat intake through diet. However, EMA states that this measure is not always feasible or sufficiently effective for reducing triglycerides and preventing pancreatitis. Traditional lipid-lowering medications have minimal impact on triglyceride levels in patients with this disease.

Redemplo contains plozasiran, a treatment based on small interfering RNA (siRNA), designed to block the production of APOC3, a protein that slows down the breakdown of fats. By blocking this protein, the medication reduces triglyceride levels in the blood and fat accumulation in the body, which is expected to reduce the risk of pancreatitis.

The medication is administered once every three months via subcutaneous injection. EMA states that Redemplo does not require genetic confirmation of the disease, unlike other authorized medications that may help individuals with genetically confirmed FCS. This difference may provide a treatment option for more adults diagnosed with FCS and may address an unmet medical need.

The EMA recommendation is based on data from a main study that included 75 adults with FCS. All patients followed a controlled diet and received either Redemplo or placebo.

After 10 months of treatment, patients who received Redemplo had an average reduction of 80% in blood triglyceride levels, compared to an average reduction of 17% in the placebo group. The effect was observed in both patients with genetically confirmed FCS and those clinically diagnosed based on signs and symptoms.

EMA states that the effect was maintained over the one-year duration of the study and for at least 18 months. The study also showed a significantly lower number of cases of acute pancreatitis in patients treated with Redemplo compared to those who received placebo.

The most common reported side effects for Redemplo were hyperglycemia, headache, nausea, and injection site reactions.

The CHMP opinion will be sent to the European Commission for the adoption of a decision regarding the marketing authorization at the EU level. After the authorization is granted, decisions regarding pricing and reimbursement will be made at the level of each member state, depending on the role or use of the medication in the national health system.

Redemplo is developed by Arrowhead Pharmaceuticals Ireland Limited. The medication received orphan drug designation for the treatment of familial chylomicronemia syndrome on July 19, 2021.

After the positive opinion of the CHMP, the Committee for Orphan Medicinal Products (COMP) will assess whether the orphan drug designation should be maintained. The EMA recommendation does not equate to immediate access for patients, as the final authorization belongs to the European Commission, and actual access subsequently depends on national decisions regarding pricing and reimbursement.

https://2eu.brussels/ro/stiri/agentia-europeana-pentru-medicamente-recomanda-un-nou-medicament-pentru-adultii-cu-sindrom-de-chilomicronemie-familiala