ARWR: REDEMPLO® Approved by FDA for the Treatment of FCS…

By David Bautz, PhD

NASDAQ:ARWR

READ THE FULL ARWR RESEARCH REPORT

Business Update

REDEMPLO^® Approved by FDA

On November 18, 2025, Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR) announced that the U.S. Food and Drug Administration (FDA) has approved REDEMPLO (plozasiran) as a treatment for adults suffering from familial chylomicronemia syndrome (FCS). This is the first approved product utilizing Arrowhead’s TRiM™ platform. The label contains no contraindications, warnings, no necessity for genetically-confirmed FCS, the drug can be self-administered once every three months, and it shows a benign adverse event (AE) profile with the most common AEs (≥10% of REDEMPLO-treated patients) being hyperglycemia, headache, nausea, and injection site reaction.

Arrowhead has set the wholesale acquisition cost (WAC) of $60,000 per year, which was very close to the $50,000 per year we estimated in our model. The company has decided on a pricing model that sets a single, consistent price for multiple potential indications, thus, the price will not change if, for example, plozasiran is approved for severe hypertriglyceridemia (SHTG). This is in stark contrast to Ionis Pharmaceuticals TRYNGOLZA^® (olezarsen), which is approved for FCS and for which the WAC is $595,000 per year. However, Ionis has indicated a potential WAC for sHTG of $10,000 - $20,000 per year, which would be well below the WAC for REDEMPLO. We don’t view this as an impediment for Arrowhead, however, as plozasiran has already shown what we believe to be is superior triglyceride (TG) lowering with treatment only once every three months, in comparison to TRYNGOLZA’s once monthly dosing, along with a numerical decrease in cases of acute pancreatitis (AP).

In the PALISADE trial, pooled 25 mg and 50 mg plozasiran data showed 2 (8%) cases of AP compared to 5 (20%) in the control group, thus showcasing the potential pharmacoeconomic benefit of the drug. AP is the most serious potential complication of FCS and can sometimes be fatal. It is associated with frequent hospitalizations, intensive care, and surgeries that lead to a significantly diminished quality of life for patients, with costs estimated to exceed $100,000. Guidelines recommend lowering triglycerides (TGs) to below 500 mg/dL in order to reduce the risk of AP. Given how effective REDEMPLO is at reducing TG levels (-80% change from baseline vs. -17% in the placebo group in the PALISADE trial), we view the company’s price point as being somewhat aggressive but justifiable, particularly if the SHASTA-5 trial, which will be specifically looking at the effect of plozasiran on incidence of AP, is positive.

The commercialization strategy for REDEMPLO is based on three core groups of patients with elevated triglycerides: FCS patients (persistent TGs ≥ 880 mg/dL and prevalent history of AP; approximately 6500 patients in the U.S.), high-risk SHTG (TGs ≥ 880 mg/dL or ≥ 500 mg/dL with prior AP history; approximately 1 million patients), and SHTG (TGs ≥ 500 mg/dL and elevated risk of AP; approximately 2 million patients). For FCS patients, the company will be focused on four types of specialists that primarily treat FCS patients: lipidologists, endocrinologists, preventive cardiologists, and internal medicine. At $60,000/yr, the FCS market is only valued at ~$390 million, however, with approximately 3 million SHTG patients that the company is hoping to target (assuming positive results in the ongoing SHTG clinical trials), that market is a potential multi-billion-dollar opportunity.

Conclusion

We congratulate the team at Arrowhead on the first approval for the company, which has fully validated the TRiM platform and which we believe will pave the way for many additional drug approvals in the future. The company indicated that REDEMPLO will be available for patients before the end of 2025, however, it is the results of the SHASTA-3, SHASTA-4, and MUIR-3 trials that hold the most economic opportunity for the drug. We anticipate results from those studies in the third quarter of 2026, with a potential sNDA filing (assuming positive results) in the fourth quarter of 2026 and a subsequent launch in SHTG in late 2027/early 2028. We have incorporated the approval of REDEMPLO into our model, which has resulted in a slight increase in our valuation to $76 per share.

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