This section highlights FDA-related milestones and regulatory updates for drugs developed by Arrowhead Pharmaceuticals (ARWR).
Over the past two years, Arrowhead Pharmaceuticals has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
ARO-ALK7, ARO-CFB, ARO-INHBE, ARO-RAGE, plozasiran, and Zodasiran. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
ARO-ALK7 - FDA Regulatory Timeline and Events
ARO-ALK7 is a drug developed by Arrowhead Pharmaceuticals for the following indication: For the Treatment of Obesity.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- ARO-ALK7
- Announced Date:
- June 2, 2025
- Indication:
- For the Treatment of Obesity
Announcement
Arrowhead Pharmaceuticals, Inc. announced that it has dosed the first subjects in a Phase 1/2a clinical trial of ARO-ALK7, the company's investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for obesity.
AI Summary
Arrowhead Pharmaceuticals announced that it has dosed the first subjects in a Phase 1/2a clinical trial of ARO-ALK7, an investigational RNA interference therapeutic being developed as a potential treatment for obesity. ARO-ALK7 is designed to silence the ACVR1C gene, which controls the production of Activin receptor-like kinase 7 (ALK7) in fat tissue. Preclinical studies showed that reducing ALK7 levels can lower body weight and fat mass while preserving lean muscle, highlighting a promising new approach to treating obesity.
The study will evaluate both single and multiple escalating doses of ARO-ALK7 in healthy obese volunteers and obese patients with or without type 2 diabetes. Researchers are also planning to investigate combinations of ARO-ALK7 with other obesity treatments, marking an important step forward in the development of RNA-based therapies aimed at improving body composition and metabolic health.
Read Announcement- Drug:
- ARO-ALK7
- Announced Date:
- February 25, 2025
- Indication:
- For the Treatment of Obesity
Announcement
Arrowhead Pharmaceuticals, Inc announced preclinical results on ARO-ALK7, the company's investigational RNA interference (RNAi) therapeutic targeting Activin receptor-like kinase 7 (ALK7) being developed as a potential treatment for obesity.
AI Summary
Arrowhead Pharmaceuticals has announced promising preclinical results for its investigational RNA interference therapeutic, ARO-ALK7, designed to target Activin receptor-like kinase 7 (ALK7) as a potential treatment for obesity. In non-human primate studies, single subcutaneous doses of ARO-ALK7 led to dose-dependent, lasting reductions in ALK7 mRNA levels in abdominal fat, with up to a 91% knockdown at higher doses, and sustained effects observed for 12 weeks.
In diet-induced obese mice, ARO-ALK7 treatment reduced body weight gain by 40% and cut fat mass by about 50%, while lean mass was preserved. When combined with tirzepatide, the therapeutic benefits improved even further, enhancing weight and fat loss while mitigating lean mass loss. Arrowhead plans to begin a Phase 1/2a clinical trial in New Zealand in the second quarter of 2025.
Read Announcement- Drug:
- ARO-ALK7
- Announced Date:
- December 23, 2024
- Indication:
- For the Treatment of Obesity
Announcement
Arrowhead Pharmaceuticals, Inc. announced that it has dosed the first subjects in a Phase 1/2a clinical trial of ARO-INHBE, the company's investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for obesity.
AI Summary
Arrowhead Pharmaceuticals, Inc. has begun dosing the first subjects in a Phase 1/2a clinical trial for ARO-INHBE, an investigational RNA interference therapeutic developed as a potential treatment for obesity. ARO-INHBE works by targeting a pathway that signals the body to store fat in adipose tissue. In preclinical studies, the therapy not only reduced body weight and fat mass but also showed promise in preserving lean muscle mass, potentially offering an advantage over current obesity treatments.
The trial is set up in two parts: the first will test ARO-INHBE as a single-agent therapy, while the second will evaluate it in combination with tirzepatide. This study aims to assess the safety, tolerability, and effectiveness of ARO-INHBE in patients with obesity, marking an important step forward in developing RNAi-based treatments for cardiometabolic and weight-related disorders.
Read Announcement- Drug:
- ARO-ALK7
- Announced Date:
- December 3, 2024
- Indication:
- For the Treatment of Obesity
Announcement
Arrowhead Pharmaceuticals, Inc announced that it has filed a request for regulatory clearance to initiate a Phase 1/2a clinical trial of ARO-ALK7, the company's second investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for obesity.
AI Summary
Arrowhead Pharmaceuticals, Inc. has filed a request for regulatory clearance to begin a Phase 1/2a clinical trial for ARO-ALK7, its second investigational RNA interference (RNAi) therapeutic aimed at treating obesity. The study will evaluate ARO-ALK7’s safety, how the body processes it, and its potential effectiveness as a new treatment for obesity. This RNAi approach works by reducing the activity of specific genes that contribute to the condition, offering the promise of a more targeted therapy. Initiating this trial is a key step for Arrowhead as it continues to explore innovative methods to tackle the growing health challenge of obesity. The company’s decision to advance ARO-ALK7 through clinical development reflects its ongoing commitment to developing cutting-edge treatments that address unmet medical needs.
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ARO-CFB - FDA Regulatory Timeline and Events
ARO-CFB is a drug developed by Arrowhead Pharmaceuticals for the following indication: Treatment of Complement Mediated Diseases.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- ARO-CFB
- Announced Date:
- December 11, 2024
- Indication:
- Treatment of Complement Mediated Diseases
Announcement
Arrowhead Pharmaceuticals, Inc announced interim results from a Phase 1/2a clinical study of ARO-CFB, the company's investigational RNA interference (RNAi) therapeutic targeting complement factor B being developed as a potential treatment for complement mediated diseases.
AI Summary
Arrowhead Pharmaceuticals recently announced encouraging interim results from a Phase 1/2a clinical study of ARO-CFB, an investigational RNA interference (RNAi) therapeutic. The treatment targets complement factor B, a key protein that helps drive the alternative complement pathway, which is involved in several complement-mediated diseases.
In the study, healthy volunteers showed dose-dependent reductions in circulating complement factor B levels by up to 90%. ARO-CFB also achieved near complete inhibition of both hemolytic activity and alternative pathway activity, with effects lasting more than three months after dosing. These strong pharmacodynamic results support the potential of ARO-CFB as a future treatment option, especially for patients with conditions such as immunoglobulin A nephropathy, the most common glomerular kidney disease. The investigational therapy was generally well tolerated, further bolstering optimism for its continued clinical development.
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ARO-INHBE - FDA Regulatory Timeline and Events
ARO-INHBE is a drug developed by Arrowhead Pharmaceuticals for the following indication: For the treatment of obesity and metabolic diseases.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- ARO-INHBE
- Announced Date:
- September 23, 2024
- Indication:
- For the treatment of obesity and metabolic diseases
Announcement
Arrowhead Pharmaceuticals, Inc announced that it has filed for regulatory clearance to initiate a Phase 1/2a clinical trial of ARO-INHBE, the company's investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for obesity.
AI Summary
Arrowhead Pharmaceuticals announced that it has filed for regulatory clearance to begin a Phase 1/2a clinical trial of its investigational RNA interference (RNAi) therapeutic, ARO-INHBE, being developed as a potential treatment for obesity. Preclinical studies show that ARO-INHBE reduced body weight and fat mass while potentially preserving lean muscle, offering a novel mechanism compared to current obesity therapies. The upcoming trial is designed in two parts: Part 1 will assess single and multiple doses of ARO-INHBE as a monotherapy in patients with obesity, while Part 2 will evaluate its use in combination with tirzepatide in both diabetic and non-diabetic obese patients. The therapy targets the INHBE gene, aiming to lower Activin E levels, which is believed to help improve energy balance and reduce fat accumulation, paving the way for new strategies in obesity treatment.
Read Announcement- Drug:
- ARO-INHBE
- Announced Date:
- June 24, 2024
- Indication:
- For the treatment of obesity and metabolic diseases
Announcement
Arrowhead Pharmaceuticals, Inc announced that preclinical data on ARO-INHBE, an investigational RNAi-based medicine for the treatment of obesity and metabolic diseases, were presented at the American Diabetes Association (ADA) 84th Scientific Sessions, which were held June 21-24, in Orlando, FL, and virtually.
AI Summary
Arrowhead Pharmaceuticals recently presented preclinical findings on its RNAi-based medicine ARO‐INHBE at the American Diabetes Association (ADA) 84th Scientific Sessions held in Orlando, FL, and online. The data showed that ARO‐INHBE effectively reduced the liver’s production of the INHBE gene, achieving a strong 95% reduction in its mRNA expression. This gene target is linked through extensive genetic studies to a healthier metabolic profile, as its reduction is associated with decreased abdominal fat and improved weight control.
In animal studies, treatment with ARO‐INHBE resulted in a 19% suppression in body weight gain, a 26% decrease in fat mass, while helping preserve lean mass. These encouraging results suggest that ARO‐INHBE could offer a new approach to treat obesity and metabolic diseases. Arrowhead plans to seek regulatory clearance in late 2024 to begin clinical trials, marking an important step forward in their development program.
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ARO-RAGE - FDA Regulatory Timeline and Events
ARO-RAGE is a drug developed by Arrowhead Pharmaceuticals for the following indication: Asthma.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- ARO-RAGE
- Announced Date:
- May 20, 2024
- Indication:
- Asthma
Announcement
Arrowhead Pharmaceuticals, Inc announced that new interim clinical data on ARO-RAGE, an investigational RNAi-based medicine for the treatment of inflammatory lung diseases, such as asthma, were presented at the American Thoracic Society (ATS) 2024 International Conference. Interim results from the ongoing Phase 1/2 study demonstrate that treatment with ARO-RAGE led to a reduction in soluble RAGE (sRAGE) concentration in bronchoalveolar lavage fluid (BALF) and serum in a dose-dependent manner in normal healthy volunteers (NHV) and in patients with mild to moderate asthma.
AI Summary
Arrowhead Pharmaceuticals presented new interim clinical data on ARO-RAGE at the ATS 2024 International Conference. ARO-RAGE is an investigational RNAi-based treatment for inflammatory lung diseases, including asthma. Interim Phase 1/2 study results showed that ARO-RAGE produced dose-dependent reductions in soluble RAGE (sRAGE) concentrations in bronchoalveolar lavage fluid and serum. In patients with mild to moderate asthma, two doses of ARO-RAGE reduced serum sRAGE by up to 88%, with a mean maximum reduction of 77%.
The data, gathered in both normal healthy volunteers and patients, also demonstrated long-lasting pharmacodynamic effects that support dosing every two months. These findings signal a promising path for treating lung diseases, showcasing ARO-RAGE’s potential for deep and sustained gene knockdown with a favorable safety and tolerability profile, as highlighted at the conference.
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plozasiran - FDA Regulatory Timeline and Events
plozasiran is a drug developed by Arrowhead Pharmaceuticals for the following indication: In Patients with Familial Chylomicronemia Syndrome.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- plozasiran
- Announced Date:
- June 23, 2025
- Indication:
- In Patients with Familial Chylomicronemia Syndrome
Announcement
Arrowhead Pharmaceuticals, Inc. announced that it has completed enrollment in SHASTA-3, SHASTA-4, and MUIR-3, the company's global Phase 3 clinical studies designed to support regulatory submissions for marketing approval of investigational plozasiran in the treatment of severe hypertriglyceridemia.
AI Summary
Arrowhead Pharmaceuticals, Inc. announced that it has completed enrollment in three global Phase 3 studies—SHASTA-3, SHASTA-4, and MUIR-3—to support regulatory submissions for marketing approval of its investigational RNAi therapeutic, plozasiran, for treating severe hypertriglyceridemia. These studies are designed to evaluate plozasiran’s efficacy and safety, with a focus on reducing triglyceride levels at 12 months. Completion of the primary parts of these trials is anticipated by mid-2026, followed by a topline data readout and subsequent regulatory submissions. The enrollment phase included nearly 2,200 patients conducted across 24 countries. This milestone is an important step toward building a comprehensive dataset that could provide a pathway for global regulatory submissions and potentially offer a new treatment option for patients suffering from this serious condition.
Read Announcement- Drug:
- plozasiran
- Announced Date:
- January 17, 2025
- Indication:
- In Patients with Familial Chylomicronemia Syndrome
Announcement
Arrowhead Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for investigational plozasiran for the treatment of familial chylomicronemia syndrome (FCS), a severe and rare genetic disease. T
AI Summary
Arrowhead Pharmaceuticals recently announced that the FDA has accepted its New Drug Application for plozasiran, an investigational treatment for familial chylomicronemia syndrome (FCS). FCS is a severe, rare genetic disorder that causes extremely high triglyceride levels and increases the risk of acute pancreatitis and other complications affecting quality of life. The acceptance of the NDA follows positive results from the Phase 3 PALISADE study, which showed significant reductions in triglycerides and a lower risk of acute pancreatitis in patients treated with plozasiran. With a PDUFA action date set for November 18, 2025, Arrowhead is focused on a swift and successful commercial launch pending further review. The company plans to submit additional applications for plozasiran to other regulatory agencies within 2025 to help meet the unmet medical need in FCS and related severe lipid disorders.
Read Announcement- Drug:
- plozasiran
- Announced Date:
- November 18, 2024
- Indication:
- In Patients with Familial Chylomicronemia Syndrome
Announcement
Arrowhead Pharmaceuticals, Inc announced that it submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for investigational plozasiran for the treatment of familial chylomicronemia syndrome (FCS), a severe and rare genetic disease which currently has no FDA approved treatments.
AI Summary
Arrowhead Pharmaceuticals, Inc. has taken a significant step in addressing familial chylomicronemia syndrome (FCS) by submitting a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA). The application is for investigational plozasiran, a treatment aimed at reducing dangerously high triglyceride levels that can lead to acute pancreatitis and other complications.
Currently, there are no FDA-approved therapies for FCS, a rare genetic disease that severely impacts quality of life. The NDA submission is supported by the promising results from the Phase 3 PALISADE study, which demonstrated significant reductions in triglycerides and a lower incidence of acute pancreatitis. This milestone highlights Arrowhead’s commitment to using its innovative RNA interference technology to deliver new treatment options for patients in urgent need.
Read Announcement- Drug:
- plozasiran
- Announced Date:
- November 18, 2024
- Indication:
- In Patients with Familial Chylomicronemia Syndrome
Announcement
Arrowhead also intends to submit applications for approval of investigational plozasiran for the treatment of patients with FCS to additional regulatory authorities in 2025.
AI Summary
Arrowhead Pharmaceuticals is moving forward to address the needs of patients with familial chylomicronemia syndrome (FCS), a rare genetic disorder that causes dangerously high triglyceride levels and increases the risk of acute pancreatitis. The company recently submitted a New Drug Application to the FDA for its investigational RNA interference therapeutic, plozasiran, which is designed to reduce levels of APOC3 and lower triglycerides. Positive results from the Phase 3 PALISADE study support the drug’s potential to significantly reduce both triglyceride levels and the occurrence of acute pancreatitis. Additionally, Arrowhead plans to submit approval applications for plozasiran to other regulatory authorities in 2025. This step highlights Arrowhead’s commitment to delivering innovative treatment options by using its targeted RNA interference platform to tackle serious health challenges like FCS.
Read Announcement- Drug:
- plozasiran
- Announced Date:
- November 18, 2024
- Indication:
- In Patients with Familial Chylomicronemia Syndrome
Announcement
Arrowhead Pharmaceuticals, Inc announced new results from the Phase 3 PALISADE study and the open-label extension from the Phase 2 MUIR and SHASTA-2 studies of investigational plozasiran.
AI Summary
Arrowhead Pharmaceuticals recently announced promising new results from its investigative studies on plozasiran. In the Phase 3 PALISADE study, patients with familial chylomicronemia syndrome—whether genetically confirmed or clinically diagnosed—experienced deep, sustained reductions in triglyceride levels along with improvements in several lipoproteins linked to atherosclerotic cardiovascular disease. These robust responses were observed regardless of genetic variations causing the disorder.
Moreover, the open‐label extension data from the Phase 2 MUIR and SHASTA-2 studies further support these findings. Patients with elevated triglycerides saw mean reductions of up to 73% and 86% in triglyceride levels over 15 months, coupled with positive changes in remnant and non-HDL cholesterol levels. Overall, these results underscore plozasiran’s potential as a promising therapeutic option for individuals suffering from conditions such as familial chylomicronemia syndrome, severe hypertriglyceridemia, and mixed hyperlipidemia.
Read Announcement- Drug:
- plozasiran
- Announced Date:
- September 10, 2024
- Indication:
- In Patients with Familial Chylomicronemia Syndrome
Announcement
Arrowhead Pharmaceuticals, Inc. announced that the United States Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to investigational plozasiran as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS), a severe and rare genetic disease characterized by extremely high triglyceride levels which can cause acute and potentially fatal pancreatitis.
AI Summary
Arrowhead Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to plozasiran. This investigational drug is designed to work as an adjunct to diet to help reduce triglyceride levels in adults with familial chylomicronemia syndrome (FCS). FCS is a rare and serious genetic disorder marked by extremely high triglyceride levels that can lead to acute pancreatitis, a potentially fatal condition. With no FDA-approved treatments currently available in the U.S. for FCS, Arrowhead’s announcement represents a significant step forward in addressing an unmet medical need. The Breakthrough Therapy designation is expected to help speed up the development and review process of plozasiran, ensuring that this new therapy can reach patients more quickly if its benefits continue to be supported by clinical evidence.
Read Announcement- Drug:
- plozasiran
- Announced Date:
- September 2, 2024
- Indication:
- In Patients with Familial Chylomicronemia Syndrome
Announcement
Arrowhead Pharmaceuticals, Inc. announced results from the Phase 3 PALISADE study of investigational plozasiran in patients with familial chylomicronemia syndrome (FCS), a severe and rare genetic disease which currently has no approved treatments in the U.S.
AI Summary
Arrowhead Pharmaceuticals, Inc. announced promising results from the Phase 3 PALISADE study of investigational plozasiran in patients with familial chylomicronemia syndrome (FCS), a rare and severe genetic disorder with no approved treatments in the U.S. The study showed that plozasiran reduced triglycerides by 80% from baseline and lowered the risk of developing acute pancreatitis by 83%. These significant improvements were seen in both patients with genetically confirmed FCS and those with a clinical diagnosis. The encouraging data has motivated Arrowhead to plan a New Drug Application submission to the FDA by the end of 2024. The study findings, which were also published in The New England Journal of Medicine, suggest that plozasiran could become a best-in-class treatment option for a condition that currently has very limited management choices and significant health risks.
Read Announcement- Drug:
- plozasiran
- Announced Date:
- August 21, 2024
- Indication:
- In Patients with Familial Chylomicronemia Syndrome
Announcement
Arrowhead Pharmaceuticals, Inc. announced that it will present new Phase 3 clinical data from the PALISADE study of plozasiran (ARO-APOC3) at the European Society of Cardiology (ESC) Congress 2024, being held in London August 30 through September 2, 2024.
AI Summary
Arrowhead Pharmaceuticals, Inc. announced it will present new Phase 3 data from the PALISADE study of plozasiran (ARO-APOC3) at the European Society of Cardiology (ESC) Congress 2024 in London, held from August 30 to September 2, 2024. The presentation, set for September 2, 2024 at 11:36 am BST, focuses on a Phase 3 study evaluating the efficacy and safety of the drug in adults with familial chylomicronemia syndrome. Professor Gerald Watts from the University of Western Australia will present the data during the session on small trials, trial updates, and other lipid therapy studies.
Following the ESC Congress, Arrowhead will host a virtual analyst and investor event on September 3, 2024, at 8:00 am EDT. This event will feature an encore presentation of the ESC data along with additional discussion from Arrowhead management.
Read Announcement- Drug:
- plozasiran
- Announced Date:
- June 3, 2024
- Indication:
- In Patients with Familial Chylomicronemia Syndrome
Announcement
Arrowhead Pharmaceuticals, Inc. announced topline results from the pivotal Phase 3 PALISADE study of investigational plozasiran in patients with genetically confirmed or clinically diagnosed familial chylomicronemia syndrome (FCS), a severe genetic disease with significant unmet need and no FDA approved therapies. PALISADE successfully met the primary endpoint of lowering triglycerides and met all key secondary endpoints, including reducing the incidence of acute pancreatitis compared to placebo.
AI Summary
Arrowhead Pharmaceuticals announced positive topline results from the pivotal Phase 3 PALISADE study of plozasiran for patients with familial chylomicronemia syndrome (FCS). FCS is a serious genetic condition with high triglyceride levels and no FDA-approved treatments. In the study, plozasiran showed strong performance by meeting its primary endpoint of reducing triglycerides by up to 80% compared to placebo. The results also included key secondary endpoints, such as a significant reduction in the occurrence of acute pancreatitis.
This is the company’s first investigational RNAi-based therapy to demonstrate clinical efficacy in a Phase 3 study. Additionally, plozasiran led to mean reductions in APOC3 of up to 94% at month 10. Arrowhead plans to present these findings further at its June 25, 2024, Cardiometabolic event during the Summer Series of R&D Webinars.
Read Announcement
Zodasiran - FDA Regulatory Timeline and Events
Zodasiran is a drug developed by Arrowhead Pharmaceuticals for the following indication: For the Treatment of Homozygous Familial Hypercholesterolemia.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Zodasiran
- Announced Date:
- July 8, 2025
- Indication:
- For the Treatment of Homozygous Familial Hypercholesterolemia
Announcement
Arrowhead Pharmaceuticals, Inc. announced that it has dosed the first subject in the YOSEMITE Phase 3 clinical trial of zodasiran, the company's investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for homozygous familial hypercholesterolemia (HoFH), a rare genetic condition that leads to severely elevated LDL-cholesterol and early onset cardiovascular disease.
AI Summary
Arrowhead Pharmaceuticals has begun dosing the first patient in its YOSEMITE Phase 3 clinical trial for zodasiran. This new investigational RNA interference (RNAi) therapy is being developed to treat homozygous familial hypercholesterolemia (HoFH), a rare genetic condition that causes extremely high LDL cholesterol levels and early heart disease. Zodasiran works by targeting ANGPTL3, a protein involved in lipid metabolism, offering a novel approach compared to traditional cholesterol-lowering treatments. The trial will enroll around 60 patients, including both adolescents and adults, who are already on their maximum tolerated lipid-lowering therapies. Researchers will monitor cholesterol and related lipid levels over a 12-month period to assess both the safety and efficacy of zodasiran in reducing harmful cholesterol levels in HoFH patients.
Read Announcement
