Arrowhead Pharmaceuticals is on the verge of its first European commercial launch after regulatory advisors recommended the approval of its siRNA therapy, REDEMPLO, for a rare and potentially life-threatening lipid disorder.
Arrowhead Pharmaceuticals (NASDAQ:ARWR) announced Friday that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for REDEMPLO® (plozasiran).
The committee recommended the drug as an adjunct to diet for adult patients with familial chylomicronemia syndrome (FCS), regardless of whether the condition is genetically confirmed or clinically diagnosed.
A final decision from the European Commission is anticipated in the second quarter of 2026.
FCS is a rare metabolic disease affecting approximately 1 to 13 people per million.
It is characterized by extreme triglyceride elevations that can lead to acute, recurrent, and often fatal pancreatitis.
While some existing treatments require genetic confirmation, the CHMP highlighted that REDEMPLO addresses a significant unmet need by providing an option for the broader clinically diagnosed adult population.
The recommendation is supported by results from the Phase 3 PALISADE study.
Data showed that a 25 mg dose of REDEMPLO reduced triglycerides by a median of 80% compared to a 17% reduction for the placebo group.
Furthermore, the study noted significantly fewer cases of acute pancreatitis in patients treated with the therapy.
REDEMPLO is administered via subcutaneous injection once every three months, utilizing Arrowhead's proprietary Targeted RNAi Molecule (TRiM™) platform to silence the mRNA encoding apolipoprotein C-III (apoC-III).