Arrowhead Pharmaceuticals Granted Entry in the FDA STAR Pilot Program for Plozasiran in Severe Hypertriglyceridemia

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Arrowhead Pharmaceuticals Granted Entry in the FDA STAR Pilot Program for Plozasiran in Severe Hypertriglyceridemia

- FDA STAR pilot program aims to shorten the time from complete supplement submission to the action date to allow earlier patient access to therapies that address an unmet medical need

PASADENA, Calif.--(BUSINESS WIRE)--Oct. 7, 2026--Arrowhead Pharmaceuticals, Inc.(NASDAQ: ARWR) today announced that theUnited States Food and Drug Administration(FDA) has granted plozasiran entry in the Split Real Time Application Review (STAR) pilot program in connection with Arrowhead’s upcoming supplemental new drug application (sNDA) for severe hypertriglyceridemia (sHTG). SHTG is a condition that significantly increases the risk of acute pancreatitis, which is associated with repeated hospital admissions and can be fatal.

The FDA STAR pilot program aims to shorten the time from the date of a complete sNDA submission to the action date for certain supplemental indications for already-approved drugs. To be granted entry in the STAR program, FDA must determine that clinical evidence from adequate and well-controlled investigation(s) indicates that the drug may demonstrate substantial improvement on a clinically relevant endpoint(s) over available therapies.

Once accepted into the STAR program, a supplemental application qualifies for a 6-month Priority Review compared to a 10-month standard review. In addition, FDA will generally target an “expedited review” for efficacy supplements in the STAR program, meaning an action at least 1 month before the Priority Review goal date is possible. The use of a Priority Review Voucher (PRV) is not necessary for programs that are granted entry in the STAR program, so Arrowhead retains the PRV previously purchased inAugust 2026.

“We are committed to pursuing every potential opportunity to accelerate the process of bringing plozasiran to patients, and the FDA STAR Pilot program provides important benefits to sponsors with efficacy supplements,” saidChristopher Anzalone, Ph.D., President and CEO at Arrowhead. “In addition, earning Priority Review for plozasiran without the need to use a PRV gives us the option to redeploy our voucher for a future application or monetize it.”

Arrowhead is on schedule to submit a sNDA for plozasiran in sHTG to the FDA by year-end 2026 and plans to seek regulatory approval with additional global regulatory authorities thereafter. Plozasiran has received regulatory approval, as REDEMPLO®, inthe United States, the European Union,China,Australia, andCanadaas an adjunct to diet to reduce triglycerides in adults with genetically confirmed or clinically diagnosed familial chylomicronemia syndrome (FCS), which is the most severe form of sHTG.

Severe hypertriglyceridemia (sHTG) is characterized by triglyceride (TG) levels greater than 500 mg/dL, with the most severe form being familial chylomicronemia syndrome (FCS) where TGs typically exceed 880 mg/dL. SHTG significantly increases the risk of acute pancreatitis (AP), which can often include recurrent attacks requiring repeat hospital admissions and worsening outcomes. AP risk is proportional to the number, characteristics, and concentration of triglyceride rich lipoproteins (TRLs), particularly chylomicrons, and increases as TGs rise. Elevated TGs can also increase the risk of atherosclerotic cardiovascular disease (ASCVD). Limited treatment options exist to sustainably reduce TGs below guideline directed risk thresholds.

REDEMPLO (plozasiran) is currently approved by theU.S. Food and Drug Administration,Health Canada, China’sNational Medical Products Administration, theAustralian Therapeutic Goods Administration, and by theEuropean Commissionas an adjunct to diet to reduce triglycerides for adults withFCS. REDEMPLO is the first and only siRNA treatment approved in these countries to be studied in both clinically diagnosed and genetically confirmed patients living withFCS.

REDEMPLO is designed to suppress the production of apolipoprotein C-III (APOC3), a protein produced in the liver that raises triglyceride levels by slowing their breakdown and clearance. By targeting APOC3 with sustained silencing, REDEMPLO delivers significant reductions in triglyceride levels. REDEMPLO is self-administered via subcutaneous injection once every three months.

REDEMPLO has been granted Orphan Medicinal Product Designation by the EMA for the treatment of patients withFCS, and Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation by theU.S. FDA for the treatment of patients withFCSand was also granted Breakthrough Therapy designation by theU.S. FDA in severe hypertriglyceridemia.

Sanofi acquired the rights to develop and commercialize REDEMPLO inGreater China, with Arrowhead retaining rights to REDEMPLO in all geographies, outside ofGreater China.

For more information about REDEMPLO, visitOur Medicines.

Arrowhead Pharmaceuticals (NASDAQ: ARWR) is a commercial-stage pharmaceutical company developing medicines that treat intractable diseases by silencing the genes that cause them, harnessing the natural RNA interference (RNAi) mechanism. The company has built a broad portfolio of clinical and commercial RNAi therapeutics through its industry-leading targeted RNAi molecule (TRiM™) platform, which can precisely silence genes in a wide range of cell types, including liver, lung, muscle, adipose, and central nervous system tissue. At Arrowhead, we rapidly advance potential best- and first-in-class RNAi treatments for diseases with significant unmet medical need, because every day matters to the patients we serve.

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Arrowhead Pharmaceuticals, Inc.Vince Anzalone, CFA+1 626-304-3400ir@arrowheadpharma.com

Paul Graves+1 626-304-3400media@arrowheadpharma.com

Source: Arrowhead Pharmaceuticals, Inc.