Arrowhead wins first FDA approval, igniting biotech rivalry with Ionis

The FDA approved Arrowhead Pharmaceuticals’ first drug, Redemplo, for treating familial chylomicronemia syndrome (FCS), marking the company’s shift into commercial operations after over 20 years in development.

Why it matters: This approval positions Arrowhead as a direct competitor to Ionis Pharmaceuticals in the rare disease space and sets up a larger battle in the broader triglyceride treatment market, where both companies have high stakes.

Backstory: Arrowhead specializes in RNA interference (RNAi) therapies and has long trailed behind peers like Alnylam in getting a product to market. Redemplo is a quarterly injection that showed superior efficacy and safety in trials compared to Ionis’ Tryngolza, the only other FDA-approved treatment for FCS.

Big picture: Arrowhead is pricing Redemplo at $60,000 per year, a tenth of Tryngolza’s list price, in a strategic move to build long-term market share as it eyes larger indications like severe hypertriglyceridemia. Ionis’ drug also carries a safety warning that Redemplo doesn’t have.

Zoom in: Redemplo cut triglycerides by ~70% vs. 59% for Tryngolza in Phase 3 trials and could do a lot to help the ~6,500 people afflicted with FCS in the US alone.

What’s next: Despite Arrowhead and Ionis being in an ongoing patent dispute, Redemplo will still be available by year’s end. Future competition looms in the severe hypertriglyceridemia market, with Arrowhead expected to release trial results next year.