CHMP Issues Positive Opinion for Redemplo in Adult FCS

Key Takeaways

• The CHMP adopted a positive opinion recommending REDEMPLO for adults with familial chylomicronemia syndrome as an adjunct to diet to reduce triglyceride levels.
• In PALISADE, lower triglycerides and fewer acute pancreatitis cases were reported in the REDEMPLO groups than with placebo.
• REDEMPLO is described as an apoC-III-targeted siRNA medicine given by self-administered subcutaneous injection once every three months, and a Commission decision is expected in the second quarter of 2026.

Arrowhead said EMA’s CHMP positive opinion for REDEMPLO (plozasiran) recommended the drug for adults with familial chylomicronemia syndrome as an adjunct to diet to reduce triglyceride levels.

The company linked the recommendation to PALISADE data showing a median 80% triglyceride reduction from baseline with 25 mg plozasiran, versus 17% with placebo. The recommendation was limited to adults with FCS rather than a broader triglyceride population. A European Commission decision is still pending, so the opinion does not yet constitute European approval.

PALISADE was a Phase 3 randomized, double-blind, placebo-controlled trial in adults with clinically diagnosed or genetically confirmed FCS. The study randomized 75 subjects across 39 sites in 18 countries, keeping the focus on a narrowly defined adult FCS population. Arrowhead said FCS affects an estimated 1 to 13 people per million globally and is associated with acute, recurrent, and potentially fatal pancreatitis. That disease burden aligns with the opinion’s focus on triglyceride reduction in adults with FCS.

According to the company, PALISADE met its primary endpoint and all multiplicity-controlled key secondary endpoints. The company also said the pooled dose groups showed significant reductions in triglycerides and apoC-III. In the Phase 3 PALISADE trial, median triglycerides fell 80% from baseline with 25 mg REDEMPLO and 17% with placebo. Arrowhead also said significantly fewer acute pancreatitis cases were reported in the REDEMPLO groups than in the placebo group. These findings were presented as support for the CHMP recommendation.

Arrowhead described REDEMPLO as a small interfering RNA medicine designed to silence apoC-III mRNA. The release said apoC-III is a key regulator of triglyceride metabolism that inhibits triglyceride catabolism and clearance. REDEMPLO is described as being self-administered by subcutaneous injection once every three months. The most common adverse reactions listed were hyperglycaemia, headache, nausea, and injection site reaction, with frequencies of 12.8%, 6.8%, 4.7%, and 4.7%, respectively. The release listed these safety findings without further interpretation.

The European Commission is expected to issue a decision on the Marketing Authorization in the second quarter of 2026. If approved, Arrowhead said REDEMPLO would be the first and only siRNA medicine authorized in the European Union for genetically confirmed and clinically diagnosed adults with FCS. The release also noted EMA Orphan Designation for the treatment of patients with FCS. The CHMP opinion is an intermediate European step rather than a final authorization. The next formal step is the European Commission decision expected in the second quarter of 2026.