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
plozasiran, ARO-DIMER-PA, ARO-ALK7, ARO-MAPT, Zodasiran, ARO-CFB, and ARO-INHBE. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
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:
- May 1, 2026
- Indication:
- In Patients with Familial Chylomicronemia Syndrome
Announcement
Arrowhead Pharmaceuticals, Inc. announced that the Australian Therapeutic Goods Administration (TGA) has approved REDEMPLO® (plozasiran), a small interfering RNA (siRNA) medicine, as an adjunct to diet to reduce triglyceride levels for adult patients with familial chylomicronemia syndrome (FCS) for whom standard triglyceride lowering therapies have been inadequate.
AI Summary
Arrowhead Pharmaceuticals announced that Australia’s Therapeutic Goods Administration has approved REDEMPLO (plozasiran), an siRNA medicine, as an adjunct to diet to reduce triglyceride levels in adults with familial chylomicronemia syndrome (FCS) for whom standard triglyceride-lowering therapies have been inadequate. REDEMPLO is the first and only medicine approved for FCS in Australia and is approved for genetically confirmed and clinically diagnosed adult patients.
People with FCS have very high triglycerides and a much higher risk of acute pancreatitis and long-term complications, often lowering quality of life. The approval was based on the Phase 3 PALISADE study, where REDEMPLO reduced triglycerides by a median 80% from baseline and significantly lowered acute pancreatitis versus placebo. The most common adverse reaction is hyperglycaemia (12.8%); other common reactions include headache (6.8%), nausea (4.7%), and injection-site reaction (4.7%). The TGA reviewed the application via a priority review pathway.
Read Announcement- Drug:
- plozasiran
- Announced Date:
- April 24, 2026
- Indication:
- In Patients with Familial Chylomicronemia Syndrome
Announcement
Arrowhead Pharmaceuticals, Inc. announced that the European Medicines Agency's Committee for Medicinal Products for Human Use (EMA CHMP) has adopted a positive opinion, recommending the approval of REDEMPLO® (plozasiran), a small interfering RNA (siRNA) medicine, as an adjunct to diet to reduce triglyceride levels in adult patients with familial chylomicronemia syndrome (FCS).
AI Summary
Arrowhead announced EMA's Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending approval of REDEMPLO (plozasiran), an siRNA medicine, as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). If confirmed by the European Commission, REDEMPLO would be the first and only siRNA authorized in the EU for both genetically confirmed and clinically diagnosed adult FCS patients.
FCS causes extremely high triglycerides and raises the risk of acute pancreatitis and long-term complications. The CHMP opinion relied on Phase 3 PALISADE results: REDEMPLO produced a median 80% triglyceride reduction from baseline and significantly fewer acute pancreatitis cases versus placebo.
EMA noted REDEMPLO does not require genetic confirmation, potentially widening treatment access. Common side effects include hyperglycaemia (12.8%), headache (6.8%), nausea (4.7%), and injection site reaction (4.7%). REDEMPLO has been granted Orphan Designation by the EMA.
Read Announcement- Drug:
- plozasiran
- Announced Date:
- March 28, 2026
- Indication:
- In Patients with Familial Chylomicronemia Syndrome
Announcement
Arrowhead Pharmaceuticals, Inc. announced long-term efficacy and safety data from a two-year long open-label extension (OLE) study of investigational plozasiran supporting its potential as therapeutic solution for a diverse spectrum of patients with hypertriglyceridemia (HTG).
AI Summary
Arrowhead Pharmaceuticals reported two‑year open‑label extension (Phase 2b OLE) data for investigational plozasiran showing large, durable triglyceride reductions across patients with hypertriglyceridemia. Patients with severe HTG had an 83% median drop in triglycerides, and 96% achieved levels below 500 mg/dL, a threshold linked to higher risk of acute pancreatitis. Patients from the MUIR group (mixed HTG) saw a 67% median reduction.
No adjudicated acute pancreatitis events occurred in any patient treated with plozasiran during the two‑year study. The drug also produced sustained, favorable improvements in atherogenic lipoproteins, including remnant cholesterol, non‑HDL cholesterol, and ApoB. The safety profile was consistent with earlier trials and showed no new safety signals in the OLE.
Arrowhead says these long‑term results support plozasiran’s potential as a therapeutic option for a diverse spectrum of HTG patients. Plozasiran is being advanced in ongoing Phase 3 studies.
Read Announcement- Drug:
- plozasiran
- Announced Date:
- January 7, 2026
- Indication:
- In Patients with Familial Chylomicronemia Syndrome
Announcement
Arrowhead Pharmaceuticals, Inc announced that the Chinese National Medical Products Administration (NMPA) has approved REDEMPLO® (plozasiran) for the reduction of triglyceride levels in adult patients with familial chylomicronemia syndrome (FCS).
AI Summary
Arrowhead Pharmaceuticals announced that China’s National Medical Products Administration (NMPA) has approved REDEMPLO® (plozasiran) to reduce triglyceride levels in adults with familial chylomicronemia syndrome (FCS). FCS is a rare, serious disorder that can cause extremely high triglycerides and raises the risk of acute, potentially life‑threatening pancreatitis. Sanofi will launch and market REDEMPLO in Greater China under a licensing agreement.
The NMPA approval triggers a $10 million milestone payment from Sanofi to Visirna Therapeutics, a majority‑owned Arrowhead subsidiary set up to develop and commercialize four cardiometabolic candidates in Greater China. Sanofi acquired the Greater China rights from Visirna in 2025 as part of that arrangement.
REDEMPLO is an siRNA therapy that targets apoC‑III to lower triglycerides. It is designed for subcutaneous self‑administration once every three months, giving patients a less frequent dosing option. Arrowhead and Visirna expect the approval to support patient access and ongoing commercialization efforts in China.
Read Announcement- Drug:
- plozasiran
- Announced Date:
- January 5, 2026
- Indication:
- In Patients with Familial Chylomicronemia Syndrome
Announcement
Arrowhead Pharmaceuticals, Inc. announced that Health Canada has issued a Notice of Compliance (NOC) authorizing REDEMPLO™ (plozasiran), a small interfering RNA (siRNA) medicine, as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS) for whom standard triglyceride lowering therapies have been inadequate.
AI Summary
Health Canada has issued a Notice of Compliance for REDEMPLO (plozasiran), an siRNA medicine approved as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS) when standard triglyceride‑lowering therapies are inadequate. REDEMPLO is the first and only Health Canada‑approved siRNA studied in genetically and clinically diagnosed FCS and is self‑administered by subcutaneous injection once every three months.
The approval was supported by the Phase 3 PALISADE study, a randomized, double‑blind, placebo‑controlled trial across 39 sites including five in Canada. In PALISADE, 25 mg REDEMPLO produced a median triglyceride reduction of −80% from baseline versus −17% in pooled placebo and showed a lower numerical incidence of acute pancreatitis over 12 months. The most common adverse reactions were hyperglycemia, headache, nausea, and injection‑site reactions.
FCS is a rare, severe disease with very high triglyceride levels and a greatly increased risk of acute pancreatitis. REDEMPLO offers a durable, at‑home treatment option that may help improve outcomes and quality of life for people living with FCS in Canada.
Read Announcement- Drug:
- plozasiran
- Announced Date:
- December 2, 2025
- 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 triglyceride (TG) levels in adults with severe hypertriglyceridemia (SHTG) (TG levels greater than or equal to 500 mg/dL).
AI Summary
Arrowhead Pharmaceuticals announced that the U.S. Food and Drug Administration has granted Breakthrough Therapy designation to investigational plozasiran as an adjunct to diet to reduce triglyceride (TG) levels in adults with severe hypertriglyceridemia (SHTG), defined as TGs ≥500 mg/dL. The company noted there are few adequate treatment options for the millions living with SHTG. Breakthrough Therapy status is intended to speed development and review for drugs treating serious conditions when early evidence suggests a substantial improvement over available therapies.
Arrowhead plans to complete its SHASTA-3, SHASTA-4, and MUIR-3 Phase 3 studies by mid-2026 and aims to submit a supplemental New Drug Application by year-end 2026. Plozasiran is an RNA interference (RNAi) drug that targets apolipoprotein C-III (apoC-III) to lower triglycerides. SHASTA-3 and SHASTA-4 enrolled about 750 participants combined, and MUIR-3 enrolled about 1,450; the studies evaluate percent change in fasting TGs at 12 months after four quarterly doses.
Read Announcement- Drug:
- plozasiran
- Announced Date:
- November 18, 2025
- Indication:
- In Patients with Familial Chylomicronemia Syndrome
Announcement
Arrowhead Pharmaceuticals, Inc announced that the U.S. Food and Drug Administration (FDA) has approved REDEMPLO (plozasiran), a small interfering RNA (siRNA) medicine, as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS).
AI Summary
Arrowhead Pharmaceuticals announced FDA approval of REDEMPLO (plozasiran), an siRNA medicine, as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). REDEMPLO is the first and only FDA‑approved siRNA therapy studied in both genetically confirmed and clinically diagnosed FCS and is given by a simple subcutaneous injection once every three months. It works by silencing apoC‑III, a liver protein that raises triglyceride levels.
FCS causes extremely high triglycerides and a much higher risk of acute, recurrent pancreatitis and other complications that reduce quality of life. The approval was supported by the Phase 3 PALISADE study, where 25 mg REDEMPLO reduced median triglycerides by about 80% versus 17% with placebo and showed a lower numerical rate of acute pancreatitis. Common side effects included hyperglycemia, headache, nausea, and injection‑site reactions. REDEMPLO is expected to be available in the U.S. before year‑end. Arrowhead will host a conference call and webcast today at 1:30 p.m. ET.
Read Announcement- 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 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:
- 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:
- 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
ARO-DIMER-PA FDA Regulatory Events
ARO-DIMER-PA is a drug developed by Arrowhead Pharmaceuticals for the following indication: for the Treatment of Mixed Hyperlipidemia.
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-DIMER-PA
- Announced Date:
- January 27, 2026
- Indication:
- for the Treatment of Mixed Hyperlipidemia
Announcement
Arrowhead Pharmaceuticals, Inc. announced that it has dosed the first subjects in a Phase 1/2a clinical trial of ARO-DIMER-PA, the company's investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for atherosclerotic cardiovascular disease (ASCVD) due to mixed hyperlipidemia.
AI Summary
Arrowhead Pharmaceuticals has dosed the first subjects in a Phase 1/2a clinical trial of ARO-DIMER-PA, an investigational RNA interference (RNAi) therapy for atherosclerotic cardiovascular disease (ASCVD) due to mixed hyperlipidemia. ARO-DIMER-PA is the first clinical candidate designed to silence two genes—PCSK9 and APOC3—using a single RNAi molecule. The dual-target approach, enabled by Arrowhead’s TRiM™ delivery platform, aims to lower both LDL‑cholesterol and triglycerides, which together raise ASCVD risk.
In preclinical studies, ARO-DIMER-PA potently reduced PCSK9 and APOC3 and improved non‑HDL cholesterol, LDL‑C, and triglyceride levels in hyperlipidemic nonhuman primates. The ARO‑DIMER‑PA‑1001 study (NCT07223658) is a placebo‑controlled, dose‑escalation trial testing single and multiple doses in up to 78 adults with mixed hyperlipidemia. The trial will evaluate safety, tolerability, pharmacokinetics, pharmacodynamics, and effects on LDL‑C and triglycerides, and could inform future dual‑function RNAi therapies.
Read Announcement- Drug:
- ARO-DIMER-PA
- Announced Date:
- October 7, 2025
- Indication:
- for the Treatment of Mixed Hyperlipidemia
Announcement
Arrowhead Pharmaceuticals, Inc. announced that it has filed a request for regulatory clearance to initiate a Phase 1/2a clinical trial of ARO-DIMER-PA, the company's investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for atherosclerotic cardiovascular disease (ASCVD) due to mixed hyperlipidemia.
AI Summary
Arrowhead Pharmaceuticals announced it has filed for regulatory clearance to begin a Phase 1/2a clinical trial of ARO-DIMER-PA, its new RNA interference (RNAi) therapy for atherosclerotic cardiovascular disease in people with mixed hyperlipidemia. This filing marks the first time a single RNAi molecule is designed to target two disease-related genes at once.
ARO-DIMER-PA uses Arrowhead’s proprietary TRiM™ technology to silence both PCSK9 and APOC3 genes. In preclinical studies with dyslipidemic nonhuman primates, it strongly reduced PCSK9 and APOC3 levels and improved non-HDL cholesterol, LDL cholesterol, and triglycerides.
Arrowhead submitted its clinical trial application to the New Zealand Medicines and Medical Devices Safety Authority. If approved, up to 78 adults with mixed hyperlipidemia will join a randomized, double-blind, placebo-controlled, dose-escalation study. The trial will test safety, tolerability, drug behavior, and effects on LDL and triglyceride levels after single and multiple doses, aiming to lower residual cardiovascular risk.
Read Announcement
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:
- January 6, 2026
- Indication:
- For the Treatment of Obesity
Announcement
Arrowhead Pharmaceuticals, Inc. announced interim results from two Phase 1/2a clinical trials of ARO-INHBE and ARO-ALK7, the company's investigational RNA interference (RNAi) therapeutics being developed as potential treatments for obesity.
AI Summary
Arrowhead Pharmaceuticals reported interim Phase 1/2a results for two RNAi drug candidates targeting obesity. Results showed meaningful reductions in weight, visceral fat, total fat and liver fat, and generally favorable tolerability so far.
ARO-INHBE combined with tirzepatide achieved a mean -9.4% weight loss at week 16 versus -4.8% with tirzepatide alone. In obese patients with type 2 diabetes, the combo produced about three-fold greater reductions in visceral fat (-23.2% vs -7.4%), total fat (-15.4% vs -5.3%), and liver fat (-76.7% vs -20%). As monotherapy, ARO-INHBE produced a -9.9% visceral fat reduction after a single dose (week 16), a -15.6% placebo-adjusted visceral fat reduction after two doses (week 24), and a 3.6% increase in lean muscle. Activin E levels fell markedly (mean -85% at a 400 mg dose; max -94%). Safety signals were generally mild with no drug-related discontinuations.
ARO-ALK7 showed the first human adipocyte gene knockdown for an RNAi therapeutic, with ALK7 mRNA reduced by a mean -88% (200 mg, week 8; max -94%) and a placebo-adjusted -14.1% visceral fat reduction after one dose. A virtual webinar to discuss results is scheduled for January 6, 2026 at 11:30 am EST.
Read Announcement- 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.
Read Announcement
ARO-MAPT FDA Regulatory Events
ARO-MAPT is a drug developed by Arrowhead Pharmaceuticals for the following indication: Treatment of Alzheimer's Disease and Other Tauopathies.
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-MAPT
- Announced Date:
- September 10, 2025
- Indication:
- Treatment of Alzheimer's Disease and Other Tauopathies
Announcement
Arrowhead Pharmaceuticals, Inc announced that it has filed a request for regulatory clearance to initiate a Phase 1/2a clinical trial of ARO-MAPT, the company's investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for tauopathies including Alzheimer's disease, a progressive neurodegenerative disease characterized by cognitive and functional decline.
AI Summary
Arrowhead Pharmaceuticals has asked for regulatory clearance to start a Phase 1/2a clinical trial of its investigational RNA interference (RNAi) therapy ARO-MAPT. ARO-MAPT is designed to silence the MAPT gene, which makes the tau protein that builds up in Alzheimer’s disease and other tauopathies. In animal studies, a single subcutaneous injection of ARO-MAPT showed strong, lasting reduction of tau messenger RNA and protein throughout the central nervous system.
The company used its new Targeted RNAi Molecule (TRiM™) delivery system, which can cross the blood-brain barrier and reach deep brain regions. Arrowhead’s filing with New Zealand’s Medicines and Medical Devices Safety Authority seeks approval for a placebo-controlled, dose-escalation study. Up to 64 healthy volunteers and 48 subjects with early Alzheimer’s will receive weekly and monthly doses of ARO-MAPT or placebo.
If cleared, this trial will assess safety, tolerability, pharmacokinetics, and pharmacodynamics, laying the groundwork for future testing in people with mild cognitive impairment due to Alzheimer’s disease.
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Zodasiran FDA Regulatory 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.
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ARO-CFB FDA Regulatory 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 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 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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