In 2026, China's National Medical Products Administration (NMPA) officially approved Redemplo (plozasiran), an RNA interference drug developed by Arrowhead Pharmaceuticals, for reducing triglyceride levels in adult patients with Familial Chylomicronemia Syndrome (FCS) in addition to dietary control.
This drug had previously received approval from the US FDA and Canadian regulatory agencies, becoming one of the first siRNA rare disease therapies to be launched in multiple countries globally, with Sanofi responsible for its commercialization in Greater China.
Analysts predict that Redemplo's annual sales could reach approximately $1.4 billion by 2031, signifying that RNA-targeted therapy is moving from scientific breakthroughs to true commercial scale.
Against the backdrop of innovative therapies continuously moving from the laboratory to clinical trials and the market globally, how to ensure that breakthrough technologies truly reach patients with rare diseases has become a core issue of concern for the pharmaceutical industry chain.
As a pharmaceutical distributor committed to promoting the compliant distribution and patient accessibility of innovative drugs globally, HongKong DengYueMed continuously monitors the clinical progress of cutting-edge therapeutic technologies such as RNA and their implementation in the Asian market.
A Neglected Yet Highly Deadly Rare Disease
Familial Chylomicronemia Syndrome (FCS) is an extremely rare inherited metabolic disorder. Classical epidemiological estimates place its prevalence at approximately 1 in 1 million people. However, with the widespread availability of genetic testing, the actual reported prevalence may fluctuate between 1 in 100,000 and 1 in 1 million people across different countries.
In molecular diagnostic registry studies in regions such as the UK, FCS patients are more commonly found in minority populations, particularly those of South Asian backgrounds. Furthermore, symptoms often appear in adolescence or earlier, significantly earlier than in multifactorial hypertriglyceridemia.
The core cause of this disease is a deficiency in lipoprotein lipase or related cofactors, leading to the body's inability to effectively break down triglyceride-rich chylomicrons. This results in plasma triglyceride levels that can rise several times or even dozens of times above normal, significantly increasing the risk of recurrent acute pancreatitis.
blood color comparison before and after treatment for familial chylomicronemia syndrome
With only a few thousand confirmed cases worldwide, this disease has long remained on the periphery of treatment research. Patients typically rely on extremely low-fat diets and limited traditional lipid-lowering therapies, yet still frequently experience abdominal pain, pancreatitis, and severely impaired quality of life.
It is precisely this coexistence of extremely low prevalence and extremely high disease burden that makes FCS one of the most urgent and groundbreaking areas for innovative therapies for rare diseases.
Redemplo (plozasiran): A Key Breakthrough in RNA Interference Therapy
Redemplo (plozasiran) is a small interfering RNA (siRNA) drug targeting the APOC3 pathway, used to lower triglyceride levels in adult patients with FCS on top of dietary control. This drug is not only Arrowhead's first commercialized product but also represents a significant leap from research to clinical application of RNA interference technology in the field of rare metabolic diseases.
From a molecular mechanism perspective, plozasiran reduces the production of apolipoprotein C-III by silencing APOC3 messenger RNA expression in hepatocytes, thereby promoting the clearance of triglyceride-rich lipoproteins and lowering blood lipid levels.
plozasiran inhibits APOC3 expression and its downstream mechanisms of action
This therapeutic approach, which directly regulates the expression of pathogenic genes, demonstrates the fundamental advantage of RNA therapy over traditional lipid-lowering drugs. In the pivotal Phase III study, plozasiran significantly reduced triglyceride levels in patients, with reductions reaching nearly 80%, and showed a marked decrease in the risk of acute pancreatitis.
This means that the treatment effect is not only reflected in improvements in laboratory indicators, but also in substantial benefits at the clinical outcome level.
Continuous follow-up results further indicate that the efficacy can be maintained long-term, and no significant new safety signals have emerged. This evidence collectively supports the possibility of RNA interference therapy achieving disease-modifying treatment in extremely rare metabolic diseases.
Why can rare diseases support a "billion-dollar" market?
The number of patients with FCS is extremely small, but Redemplo's commercial potential is widely recognized. This seemingly contradictory phenomenon essentially stems from the structural changes in the pharmacoeconomics of rare diseases.
First, rare diseases typically lack alternative therapies, their clinical value is highly concentrated, and regulatory approval pathways are more certain. Previously, the United States had virtually no effective treatments for FCS, relying solely on strict dietary control and limited lipid-lowering drugs, with insufficient efficacy.
Second, RNA therapy has characteristics such as long dosing intervals and sustained effects. plozasiran, administered approximately once every 12 weeks in trials, demonstrated stable and sustained efficacy. This chronic disease management model aligns more closely with commercially viable sustainable products than with a one-time treatment.
Therefore, even with a very small patient base, high-value pricing and long-term treatment cycles can still create a substantial market, making it a significant paradigm for the commercialization of RNA drugs for rare diseases in recent years.
Competition and Expansion in the RNA Drug Market
Redemplo (plozasiran) is not the only innovative therapy targeting the APOC3 pathway.
olezarsen (Tryngolza), developed by Ionis Pharmaceuticals, received FDA approval in 2024 for familial chylomicronemia (FCS). It significantly reduced triglycerides and acute pancreatitis events on a low-fat diet. Its Phase III Balance study showed a placebo-corrected reduction of approximately 42.5% at 6 months, validating the clinical feasibility of APOC3 inhibition as a disease modification strategy.
As a small interfering RNA (siRNA) therapy delivered via GalNAc, plozasiran significantly reduces apoC-III levels and promotes the clearance of triglyceride-rich lipoproteins by silencing APOC3 mRNA in hepatocytes.
Based on evidence from randomized controlled trials, APOC3-targeting RNA drugs such as volanesorsen, olezarsen, and plozasiran consistently reduce triglycerides and pancreatitis events, demonstrating consistent clinical benefits. This suggests that RNA-targeted lipid metabolism pathways are moving from a single-point breakthrough to a platform-based treatment paradigm.
Conclusion: The Starting Point of the RNA Era
The approval of Redemplo in China signifies that RNA interference therapy has completed a crucial leap from a research tool to a commercially available treatment solution in multiple locations worldwide.
When a technology can achieve clear clinical benefits, sustainable dosing protocols, and cross-border regulatory approval in a very small patient population, it changes not only the treatment of a single disease but also the entire biopharmaceutical industry's logic for judging the value of technology. In the next decade, the areas where RNA drugs are most likely to experience explosive growth will remain concentrated in "small population, high-value" scenarios such as rare diseases and metabolic disorders.
As patients’ demand for access to innovative therapies continues to rise, the international pharmaceutical supply and distribution system of DengYue Medicine will play an increasingly crucial bridging role in the commercialization of RNA therapy.
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