Arrowhead Pharmaceuticals Secures FDA Approval for REDEMPLO® (Plozasiran) to Treat Familial Chylomicronemia Syndrome (FCS)
Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) has announced a groundbreaking milestone with the U.S. Food and Drug Administration (FDA) granting approval for REDEMPLO® (plozasiran), a small interfering RNA (siRNA) therapeutic. This innovative treatment is designed to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS), a rare and severe genetic condition. REDEMPLO is the first and only FDA-approved siRNA medicine specifically studied in patients with genetically confirmed and clinically diagnosed FCS.
FCS affects an estimated 6,500 individuals in the U.S., causing triglyceride levels to soar 10 to 100 times above normal. This condition significantly increases the risk of acute pancreatitis, chronic abdominal pain, and other long-term complications, often leading to a diminished quality of life. REDEMPLO offers a new beacon of hope for these patients, providing a convenient, self-administered subcutaneous injection every three months.
“This FDA approval is a transformational milestone for Arrowhead and a proud moment for everyone involved in the discovery and development process,” said Christopher Anzalone, Ph.D., President and CEO of Arrowhead Pharmaceuticals. “REDEMPLO represents new hope for patients living with FCS and marks the beginning of a new chapter for Arrowhead as we transition into a commercial-stage company.”
Breakthrough Results from the PALISADE Study
The FDA’s approval of REDEMPLO is based on robust data from the Phase 3 PALISADE study, a randomized, double-blind, placebo-controlled trial. The study demonstrated that REDEMPLO significantly reduced triglycerides and lowered the incidence of acute pancreatitis compared to placebo. Patients receiving 25 mg of REDEMPLO experienced a median triglyceride reduction of 80% from baseline, compared to a 17% reduction in the placebo group.
The PALISADE study also highlighted the safety profile of REDEMPLO, with the most common adverse reactions being hyperglycemia, headache, nausea, and injection site reactions. Importantly, the treatment has no contraindications, warnings, or precautions listed in its prescribing information.
A Patient-Centric Approach
Lindsey Sutton Bryan, co-founder and co-president of the FCS Foundation, emphasized the significance of this approval for the FCS community. “Today’s approval marks a pivotal moment for people living with FCS. This community has often been overlooked and misunderstood, making their journey to effective treatment especially challenging. REDEMPLO offers real hope for a better future and demonstrates the power of innovation driven by empathy and collaboration.”
Arrowhead has also launched the “Rely On REDEMPLO” patient support program, which provides resources and financial assistance to ensure access to this life-changing therapy.
A New Era for Arrowhead Pharmaceuticals
REDEMPLO is Arrowhead’s first FDA-approved medicine and is built on the company’s proprietary Targeted RNAi Molecule (TRiM™) platform. This platform enables precise gene silencing, with the potential to target multiple cell types and silence two genes simultaneously within a single molecule. The approval of REDEMPLO underscores Arrowhead’s leadership in RNAi-based therapeutics and its commitment to addressing unmet medical needs.
Arrowhead is also advancing plozasiran in additional Phase 3 studies, including SHASTA-3, SHASTA-4, SHASTA-5, and MUIR, to explore its potential in treating other lipid disorders.
Availability and Next Steps
REDEMPLO is expected to be available in the U.S. by the end of the year. The treatment has also received Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation from the FDA, as well as Orphan Medicinal Product Designation from the European Medicines Agency.
Arrowhead will host a conference call and webcast today at 1:30 p.m. ET to discuss the FDA approval and the company’s future plans. A replay of the webcast will be available on the Arrowhead website.
About Familial Chylomicronemia Syndrome (FCS)
FCS is a rare genetic disorder characterized by extremely high triglyceride levels, often exceeding 880 mg/dL. This condition can lead to severe complications, including acute pancreatitis, diabetes, and cognitive issues. Current treatment options for FCS are limited, making the approval of REDEMPLO a significant advancement in addressing this unmet medical need.
About Arrowhead Pharmaceuticals
Arrowhead Pharmaceuticals is dedicated to developing innovative medicines that silence disease-causing genes through RNA interference (RNAi). By leveraging its TRiM™ platform, Arrowhead aims to deliver rapid, deep, and durable gene silencing, transforming the treatment landscape for intractable diseases.
For more information, visit www.arrowheadpharma.com.
