FDA Approves REDEMPLO, First-of-Kind siRNA Therapy for Familial Chylomicronemia Syndrome
A groundbreaking new treatment offers hope for individuals with a rare and severe genetic disorder characterized by dangerously high triglyceride levels.
The U.S. Food and Drug governance (FDA) has approved REDEMPLO (plozasiran), a novel small interfering RNA (siRNA) medicine, as an adjunct to diet for adults with familial chylomicronemia syndrome (FCS). This approval marks a significant advancement in the treatment of this debilitating condition, offering a targeted therapy for a historically underserved patient population. FCS affects an estimated 6,500 people in the U.S., causing triglyceride levels 10 to 100 times higher then normal, substantially increasing the risk of acute, recurrent, and potentially fatal pancreatitis.
REDEMPLO is the first and only FDA-approved siRNA medicine specifically designed for FCS. The treatment is self-administered via a simple subcutaneous injection once every three months, offering a convenient and potentially life-altering option for patients. According to a company release, the medicine utilizes Arrowhead Pharmaceuticals’ proprietary Targeted RNAi Molecule (TRiM™) platform. This approval represents a major milestone for Arrowhead as it transitions into a commercial-stage biotechnology company.
“This is a transformational milestone for Arrowhead,” stated Christopher Anzalone, Ph.D., President and CEO at Arrowhead Pharmaceuticals. “This approval, and subsequent launch, marks the beginning of a new chapter in our journey-one rooted in our unwavering commitment to delivering life-changing therapies to patients with serious diseases.” he further emphasized the potential of the TRiM™ platform, noting its capability to deliver siRNA to seven diffrent cell types and potentially silence the expression of two genes simultaneously.
The FDA’s decision was supported by robust clinical data from the Phase 3 PALISADE study, a randomized, double-blind, placebo-controlled trial involving adults with genetically confirmed or clinically diagnosed FCS. The study successfully met its primary endpoint and all pre-specified secondary endpoints, demonstrating significant reductions in both triglycerides and APOC3. Specifically, patients receiving 25 mg of REDEMPLO experienced a median 80% reduction in triglycerides from baseline, compared to a 17% reduction in the placebo group.Furthermore, the study indicated a lower incidence of acute pancreatitis among those treated with REDEMPLO.
Lindsey Sutton Bryan, co-founder and co-president of the FCS Foundation, highlighted the importance of this approval for the FCS community.”today’s approval marks a pivotal moment for people living with familial chylomicronemia syndrome and the physicians who support them,” she said. “Because FCS symptoms are mostly invisible, this community historically has been often overlooked and misunderstood, making their journey to effective treatment especially difficult.”
Clinical trials revealed the most common adverse reactions in patients treated with REDEMPLO (incidence ≥10% and >5% more frequently than with placebo) to be hyperglycemia,headache,nausea,and injection site reaction. Importantly, the U.S. approved package insert lists no contraindications, warnings, or precautions associated with the use of REDEMPLO.
the compelling efficacy and safety results from the PALISADE study were presented at both the European Society of Cardiology (ESC) Congress 2024 and the American heart Association Scientific Sessions 2024 (AHA24), and simultaneously published in The New England Journal of Medicine.
Arrowhead Pharmaceuticals is dedicated to developing medicines for intractable diseases by silencing the genes that cause them. The company’s RNAi-based therapeutics leverage the natural gene silencing pathway, utilizing a broad portfolio of RNA chemistries and efficient delivery methods to induce rapid, deep, and durable knockdown of target genes.
For more information, visit www.arrowheadpharma.com or connect with Arrowhead on X (formerly Twitter) at @ArrowheadPharma, LinkedIn, Facebook, and Instagram. Interested parties can also subscribe to the company’s email list for direct news updates.
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