Arrowhead Secures Maiden FDA Approval for Redemplo in Rare Genetic Metabolic Disorder (FCS)

Arrowhead Pharmaceuticals received its first-ever FDA approval for Redemplo (plozasiran), a therapy for adults with familial chylomicronemia syndrome (FCS), a rare genetic metabolic disorder that impairs triglyceride breakdown in the bloodstream1235.

Redemplo is indicated as an adjunct to diet to reduce triglyceride levels in adults with FCS1235.

The approval is a major milestone for Arrowhead, marking its transition to a commercial-stage company after two decades of focusing on RNA interference (RNAi) drug development1234.

Redemplo was studied in genetically confirmed and clinically diagnosed FCS patients and is the first and only FDA-approved medicine specifically for this population3.

In Phase 3 trials, Redemplo achieved a 70% reduction in triglyceride levels, demonstrating superior efficacy to its rival Tryngolza (approved in 2024), which achieved a 59% reduction2.

Redemplo will launch at an annual list price of $60,000—substantially lower than Tryngolza’s $595,000 per year2.

The drug is self-administered by subcutaneous injection once every three months1.

Redemplo leverages Arrowhead’s TRiM™ (Targeted RNAi Molecule) platform, which enables precise gene silencing and is viewed as a significant technological innovation in RNAi therapies34.

Redemplo received Breakthrough Therapy, Fast Track, and Orphan Drug designations from the FDA, as well as Orphan Medicinal Product status from the European Medicines Agency5.

FCS is an ultra-rare disorder, estimated to affect about 6,500 people in the U.S.; symptoms are often invisible and have historically made effective treatment inaccessible13.

Sources:

1. https://www.biopharmadive.com/news/arrowhead-fda-approve-redemplo-familial-chylomicronemia-syndrome-ionis/805694/

2. https://trial.medpath.com/news/501415ed5ad29b30/arrowhead-pharmaceuticals-secures-first-fda-approval-with-redemplo-for-rare-genetic-disorder

3. https://arrowheadpharma.com/news-press/arrowhead-pharmaceuticals-announces-fda-approval-of-redemplo-plozasiran-to-reduce-triglycerides-in-adults-with-familial-chylomicronemia-syndrome-fcs/

4. https://www.businesswire.com/news/home/20251112678197/en/Arrowhead-Pharmaceuticals-Announces-FDA-Approval-of-REDEMPLO-plozasiran-to-Reduce-Triglycerides-in-Adults-with-Familial-Chylomicronemia-Syndrome-FCS

5. https://globalgenes.org/raredaily/fda-approves-arrowheads-redemplo-for-fcs/