- Plozasiran, an siRNA targeting APOC3, reduced triglycerides by 80% versus 17% with placebo after 10 months in a 75-adult trial.
- Administered subcutaneously every three months, the effect was maintained during the one-year study and up to at least 18 months.
- The study reported significantly fewer acute pancreatitis cases versus placebo.
- EMA's CHMP issued a positive opinion and sent it to the European Commission for an EU marketing-authorisation decision; pricing and reimbursement will be decided by member states.
EMA recommendation
EMA’s human medicines committee (CHMP) recommended granting an EU marketing authorisation for Redemplo (plozasiran) to treat adults with familial chylomicronaemia syndrome (FCS) and forwarded its opinion to the European Commission.
Disease overview
FCS is a rare inherited disorder that prevents normal lipid breakdown, causing extremely high triglycerides and symptoms including severe abdominal pain, potentially fatal acute pancreatitis, hepatosplenomegaly, diabetes, cognitive issues and xanthomas; patients rely on strict low‑fat diets that are often insufficient.
Mechanism and dosing
Plozasiran is a first‑in‑class small interfering RNA (siRNA) that blocks APOC3 production, reducing triglyceride levels and fat accumulation; it is administered by subcutaneous injection every three months.
Clinical evidence
The pivotal trial included 75 adults on a controlled diet randomized to plozasiran or placebo; after 10 months plozasiran reduced triglycerides by 80% versus 17% with placebo, with the effect maintained during the one‑year study and up to at least 18 months, and significantly fewer acute pancreatitis cases observed versus placebo.
Safety
The most common adverse events reported were hyperglycaemia, headache, nausea and injection‑site reactions.
Access considerations
Redemplo does not require genetic confirmation of FCS, potentially broadening eligible patients; if authorised, EU pricing and reimbursement decisions will be taken by individual Member States.
