This section highlights FDA-related milestones and regulatory updates for drugs developed by Alnylam Pharmaceuticals (ALNY).
Over the past two years, Alnylam Pharmaceuticals has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
Fitusiran, GIVLAARI, nucresiran, Vutrisiran, Vutrisiran, and Zilebesiran. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Fitusiran - FDA Regulatory Timeline and Events
Fitusiran is a drug developed by Alnylam Pharmaceuticals for the following indication: Hemophilia A or B.
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:
- Fitusiran
- Announced Date:
- March 28, 2025
- Indication:
- Hemophilia A or B
Announcement
Alnylam Pharmaceuticals, Inc today highlighted the significance of the U.S. Food and Drug Administration's (FDA) approval of Qfitlia™ (fitusiran), the sixth Alnylam-discovered RNAi therapeutic approved in the U.S., and the first and only therapeutic to lower antithrombin (AT), a protein that inhibits blood clotting, with the goal of promoting thrombin generation to rebalance hemostasis and prevent bleeds.
AI Summary
Alnylam Pharmaceuticals recently celebrated the U.S. FDA approval of Qfitlia™ (fitusiran), marking a major breakthrough as the sixth RNAi therapeutic discovered by the company to gain approval and the first and only one that lowers antithrombin (AT). By reducing AT, Qfitlia works to promote thrombin generation, helping to rebalance hemostasis and prevent bleeding episodes in patients with hemophilia A or B, with or without inhibitors. This unique approach sets Qfitlia apart from other treatments and offers a new non-factor option for patients. The approval highlights the drug's potential to significantly reduce the frequency of bleeds as shown in clinical studies. Patients aged 12 and older can now benefit from this innovative subcutaneous injection treatment that offers routine prophylaxis against bleeding, representing a significant advancement in hemophilia care.
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GIVLAARI - FDA Regulatory Timeline and Events
GIVLAARI is a drug developed by Alnylam Pharmaceuticals for the following indication: Acute Hepatic Porphyria (AHP).
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:
- GIVLAARI
- Announced Date:
- December 23, 2024
- Indication:
- Acute Hepatic Porphyria (AHP)
Announcement
Alnylam Canada ULC announce that GIVLAARI® (givosiran injection) is now funded across Canada through both public and private insurance plans for patients living with acute hepatic porphyria (AHP), including those diagnosed with acute intermittent porphyria (AIP) - the most common type of AHP.
AI Summary
Alnylam Canada ULC announced that GIVLAARI® (givosiran injection) is now funded across Canada through both public and private insurance plans. This means that patients with acute hepatic porphyria (AHP), including those diagnosed with acute intermittent porphyria (AIP) – the most common type – can now access this innovative treatment without financial obstacles.
GIVLAARI is the first and only therapy in Canada that has been proven to prevent AHP attacks, reduce chronic pain, and improve overall quality of life. The funding decision came after close collaboration between Alnylam and various government agencies, showing a strong commitment to supporting patients with this ultra-rare, genetic condition. This milestone provides hope for better management of AHP symptoms and a more normal daily life for affected individuals.
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nucresiran - FDA Regulatory Timeline and Events
nucresiran is a drug developed by Alnylam Pharmaceuticals for the following indication: For the treatment of transthyretin (ATTR) amyloidosis.
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:
- nucresiran
- Announced Date:
- November 17, 2024
- Estimated Event Date Range:
- January 1, 2025 - March 31, 2025
- Target Action Date:
- Q1 2025
- Indication:
- For the treatment of transthyretin (ATTR) amyloidosis
Announcement
Alnylam Pharmaceuticals, Inc announced that it Alnylam Continues to Expect to Share Phase 3 Development Plans in Q1 2025 –
AI Summary
Alnylam Pharmaceuticals announced encouraging Phase 1 data for its next-generation RNAi therapeutic, nucresiran, for treating ATTR amyloidosis. In the study, a single 300 mg dose produced rapid, robust, and sustained reductions in serum TTR levels—over 90% by Day 15, peaking at more than 96% by Day 29, and maintaining significant knockdown through six months. These results suggest that nucresiran could allow for biannual or even annual subcutaneous dosing, signaling a potential new paradigm in treating the disease.
Building on these promising findings and the favorable safety and tolerability profile, Alnylam continues to develop its approach to ATTR amyloidosis. The company expects to share its Phase 3 development plans in the first quarter of 2025, which will provide additional details and insights into the therapeutic’s future in addressing this debilitating condition.
Read Announcement- Drug:
- nucresiran
- Announced Date:
- November 17, 2024
- Indication:
- For the treatment of transthyretin (ATTR) amyloidosis
Announcement
Alnylam Pharmaceuticals, Inc. announced the presentation of new results from its Phase 1 study of nucresiran (formerly ALN-TTRsc04), a next-generation RNAi therapeutic in development for the treatment of transthyretin (ATTR) amyloidosis.
AI Summary
Alnylam Pharmaceuticals recently presented promising Phase 1 results for nucresiran (formerly ALN-TTRsc04), a next-generation RNAi therapeutic developed for ATTR amyloidosis. In an oral session at the American Heart Association Scientific Sessions 2024, the study showed that a single dose of 300 mg or higher led to a rapid reduction in serum TTR levels by more than 90% as early as Day 15, with peak reductions over 96% by Day 29. These effects were highly durable, with substantial TTR lowering maintained through at least six months and evidence of continued reduction at Day 360 for the 300 mg dose. The data also highlighted low variability in TTR knockdown among patients, supporting the potential for biannual or even annual dosing. Nucresiran was well tolerated at all doses, and Alnylam plans to announce further Phase 3 development plans in the first quarter of 2025.
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Vutrisiran - FDA Regulatory Timeline and Events
Vutrisiran is a drug developed by Alnylam Pharmaceuticals for the following indication: For the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy.
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:
- Vutrisiran
- Announced Date:
- June 9, 2025
- Indication:
- For the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy.
Announcement
Alnylam Pharmaceuticals, Inc. announced that the European Commission (EC) has granted approval for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM) as an additional indication for its orphan RNAi therapeutic AMVUTTRA® (vutrisiran).
AI Summary
Alnylam Pharmaceuticals, Inc. announced that the European Commission has approved AMVUTTRA® (vutrisiran) for treating wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). This approval makes AMVUTTRA the first RNA interference (RNAi) therapeutic in Europe targeting the cardiomyopathy manifestations of ATTR amyloidosis. The decision is based on strong results from the HELIOS-B Phase 3 study, which showed that the treatment could lower all-cause mortality and improve functional capacity and quality of life in patients. By working at the root of the disease through rapid and sustained knockdown of transthyretin (TTR), AMVUTTRA delivers a novel therapeutic approach. This milestone is expected to provide a crucial new treatment option for an estimated 100,000 Europeans living with ATTR amyloidosis, helping to address this challenging and underserved condition.
Read Announcement- Drug:
- Vutrisiran
- Announced Date:
- March 20, 2025
- Indication:
- For the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy.
Announcement
Alnylam Pharmaceuticals, Inc announced the U.S. Food and Drug Administration (FDA) approval of the supplemental New Drug Application (sNDA) for its RNAi therapeutic, AMVUTTRA® (vutrisiran), for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits.
AI Summary
Alnylam Pharmaceuticals recently announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental New Drug Application (sNDA) for its RNAi therapeutic, AMVUTTRA® (vutrisiran). This approval allows AMVUTTRA to be used for treating the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults. The therapy works by rapidly reducing the production of transthyretin (TTR), which helps lower the risk of cardiovascular mortality, hospitalizations, and urgent heart failure visits.
This decision marks a major advancement in treating ATTR-CM, as it targets the root cause of the disease with a convenient quarterly dosing schedule. The FDA’s approval of AMVUTTRA provides a new treatment option that may generate better clinical outcomes and improved quality of life for patients battling this serious and often fatal condition.
Read Announcement- Drug:
- Vutrisiran
- Announced Date:
- November 25, 2024
- Indication:
- For the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy.
Announcement
Alnylam Pharmaceuticals, Inc announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Company's supplemental New Drug Application (sNDA) for vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM).
AI Summary
Alnylam Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental New Drug Application (sNDA) for vutrisiran. This investigational RNAi therapeutic is being developed to treat ATTR amyloidosis with cardiomyopathy (ATTR-CM), a serious condition affecting the heart. The FDA’s acceptance of the sNDA is a key step in reviewing vutrisiran’s potential use for patients suffering from this progressive disease.
The review was granted using a Priority Review Voucher, and the FDA has set a target action date of March 23, 2025, under the Prescription Drug User Fee Act. Alnylam’s decision to move forward with this application follows promising results from clinical studies, offering hope for better cardiovascular outcomes and quality of life for patients in need of new treatment options.
Read Announcement- Drug:
- Vutrisiran
- Announced Date:
- November 25, 2024
- Estimated Event Date Range:
- March 23, 2025 - March 23, 2025
- Target Action Date:
- March 23, 2025
- Indication:
- For the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy.
Announcement
Alnylam Pharmaceuticals, Inc announced that the FDA has set an action date goal of March 23, 2025, under the Prescription Drug User Fee Act (PDUFA).
AI Summary
Alnylam Pharmaceuticals announced that the FDA has accepted its supplemental New Drug Application (sNDA) for vutrisiran, an investigational RNAi therapeutic for treating ATTR amyloidosis with cardiomyopathy. Using a Priority Review Voucher, the FDA has set an action date goal of March 23, 2025, under the Prescription Drug User Fee Act (PDUFA). This means that the review process will be completed by that date, bringing the decision closer for a treatment that targets both neuropathy and cardiomyopathy symptoms.
If approved, vutrisiran would become the first therapy in the U.S. to address these dual manifestations of the disease. The decision by the FDA highlights its commitment to expedite potentially life-changing treatments for patients affected by this debilitating condition. The company looks forward to further discussions with the agency as the review process unfolds.
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Vutrisiran (HELIOS-B) - FDA Regulatory Timeline and Events
Vutrisiran (HELIOS-B) is a drug developed by Alnylam Pharmaceuticals for the following indication: Transthyretin-Mediated (ATTR) Amyloidosis with Cardiomyopathy.
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:
- Vutrisiran (HELIOS-B)
- Announced Date:
- May 12, 2025
- Indication:
- Transthyretin-Mediated (ATTR) Amyloidosis with Cardiomyopathy
Announcement
Alnylam Pharmaceuticals, Inc announced that the Company will present the latest data from its flagship transthyretin amyloidosis (TTR) franchise at the upcoming Heart Failure 2025 Congress, a scientific congress of the European Society of Cardiology, taking place May 17-20 in Belgrade, Serbia.
AI Summary
Alnylam Pharmaceuticals, Inc. will present its latest data from the flagship transthyretin amyloidosis (TTR) franchise at the Heart Failure 2025 Congress. This scientific meeting, hosted by the European Society of Cardiology, will take place in Belgrade, Serbia from May 17-20, 2025. The data presentation will focus on new analyses from the HELIOS-B Phase 3 study of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy. Key findings will include extended outcomes on cardiovascular hospitalizations and urgent heart failure visits up to 42 months, providing valuable insights into the efficacy of the treatment. By sharing these results, Alnylam aims to highlight the potential of AMVUTTRA® (vutrisiran) in managing this challenging condition and reinforcing its role as a leader in RNAi therapeutics for tackling the root cause of TTR amyloidosis.
Read Announcement- Drug:
- Vutrisiran (HELIOS-B)
- Announced Date:
- April 28, 2025
- Estimated Event Date Range:
- June 1, 2025 - June 30, 2025
- Target Action Date:
- June 01, 2025
- Indication:
- Transthyretin-Mediated (ATTR) Amyloidosis with Cardiomyopathy
Announcement
Alnylam Pharmaceuticals, Inc announced that European Commission Decision Expected in June 2025
AI Summary
Alnylam Pharmaceuticals, Inc. announced that the European Commission is expected to review its RNA interference therapeutic vutrisiran in June 2025. The decision could lead to approval for treating ATTR cardiomyopathy, a serious condition caused by misfolded transthyretin protein deposits in the heart. Vutrisiran has shown promising results in the pivotal HELIOS-B Phase 3 study, where it significantly reduced mortality and cardiovascular events while preserving patients’ functional status and quality of life. The treatment is set apart by its rapid and sustained TTR knockdown and its quarterly, subcutaneous dosing, which provides convenience and flexibility. With these encouraging clinical outcomes, vutrisiran may become a valuable new option for patients suffering from this debilitating cardiac condition.
Read Announcement- Drug:
- Vutrisiran (HELIOS-B)
- Announced Date:
- April 28, 2025
- Indication:
- Transthyretin-Mediated (ATTR) Amyloidosis with Cardiomyopathy
Announcement
Alnylam Pharmaceuticals, Inc announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending approval of its RNAi therapeutic vutrisiran for the treatment of wild type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
AI Summary
Alnylam Pharmaceuticals, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has given a positive opinion for its RNAi therapeutic, vutrisiran, to treat wild type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). This recommendation follows the successful results from the pivotal HELIOS-B Phase 3 study, which showed that vutrisiran significantly reduced mortality and cardiovascular events while helping maintain patients’ functional status and quality of life.
Vutrisiran works by rapidly and sustainably reducing transthyretin (TTR) levels through quarterly subcutaneous injections. This innovative treatment offers a differentiated approach in managing a serious and often fatal condition that has limited treatment options. The positive CHMP opinion marks an important step toward European approval, potentially making vutrisiran the first and only RNAi therapeutic to be approved for ATTR-CM in the EU.
Read Announcement- Drug:
- Vutrisiran (HELIOS-B)
- Announced Date:
- March 29, 2025
- Indication:
- Transthyretin-Mediated (ATTR) Amyloidosis with Cardiomyopathy
Announcement
Alnylam Pharmaceuticals, Inc. announced moderated poster presentations of new data from the landmark HELIOS-B Phase 3 clinical trial, which evaluated vutrisiran for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM) in a population representative of today's patients.
AI Summary
Alnylam Pharmaceuticals announced moderated poster presentations showcasing new data from the landmark HELIOS‑B Phase 3 clinical trial. The trial evaluated vutrisiran for treating ATTR amyloidosis with cardiomyopathy (ATTR‑CM) in a patient group that reflects today’s real-world population. The findings highlight vutrisiran’s ability to improve cardiac function by enhancing both systolic and diastolic measures and slowing the decline in heart performance. These improvements in heart function are important as they may help reduce the risk of further heart complications. The data further showed that patients using vutrisiran maintained or improved their functional capacity, overall health, and quality of life compared to those receiving a placebo. The benefits were seen across different levels of heart failure severity, with the greatest improvements noted in patients with less severe disease at the start of treatment.
Read Announcement- Drug:
- Vutrisiran (HELIOS-B)
- Announced Date:
- October 9, 2024
- Indication:
- Transthyretin-Mediated (ATTR) Amyloidosis with Cardiomyopathy
Announcement
Alnylam Pharmaceuticals, Inc announced the submission of its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM).
AI Summary
Alnylam Pharmaceuticals, Inc. announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for vutrisiran, an investigational RNAi therapeutic for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM). The sNDA is based on positive full findings from the HELIOS-B Phase 3 study, which showed that vutrisiran can rapidly lower transthyretin (TTR) levels and improve outcomes such as reducing death and cardiovascular events while delaying disease progression. To accelerate the review process, the company used a Priority Review Voucher, which ensures a faster evaluation timeline by the FDA. Pending regulatory approval, vutrisiran could potentially become a first-line therapy for patients with ATTR-CM. The company is actively collaborating with the FDA and is planning additional filings in other regions to bring this promising treatment to market as soon as possible.
Read Announcement- Drug:
- Vutrisiran (HELIOS-B)
- Announced Date:
- September 29, 2024
- Indication:
- Transthyretin-Mediated (ATTR) Amyloidosis with Cardiomyopathy
Announcement
Alnylam Pharmaceuticals, Inc. announced that two new data sets from the HELIOS-B Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM), were presented in the Late Breaking Clinical Research Session 1 at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2024, which was held virtually.
AI Summary
Alnylam Pharmaceuticals, Inc. announced that two new data sets from the HELIOS-B Phase 3 study of vutrisiran were presented during the Late Breaking Clinical Research Session 1 at the virtual HFSA Annual Scientific Meeting 2024. Vutrisiran is an investigational RNAi therapeutic being developed to treat ATTR amyloidosis with cardiomyopathy (ATTR-CM). The presented data showed that treatment with vutrisiran improved patients’ cardiac structure and function. Specifically, improvements were seen in both systolic and diastolic parameters over 30 months, with stable levels of important cardiac biomarkers compared to placebo, suggesting a potential disease-modifying effect. Alnylam’s Chief Medical Officer highlighted that early intervention with vutrisiran could provide significant benefits for patients, and the company remains confident that, with regulatory approval, vutrisiran may soon become a first-line therapy for ATTR-CM.
Read Announcement- Drug:
- Vutrisiran (HELIOS-B)
- Announced Date:
- August 30, 2024
- Indication:
- Transthyretin-Mediated (ATTR) Amyloidosis with Cardiomyopathy
Announcement
Alnylam Pharmaceuticals, Inc announced detailed results from the HELIOS-B Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM).
AI Summary
Alnylam Pharmaceuticals announced detailed results from the HELIOS-B Phase 3 study of vutrisiran, an investigational RNAi therapeutic for ATTR amyloidosis with cardiomyopathy (ATTR-CM). In the overall population, vutrisiran achieved a 28% reduction in the composite endpoint of all-cause mortality and recurrent cardiovascular events, with reductions in mortality of 31% during the double-blind period and 36% up to 42 months. Among patients receiving vutrisiran as monotherapy, the study reported a 33% reduction in the primary endpoint and a 35% reduction in all-cause mortality up to 42 months.
The study also showed strong trends of additive efficacy when used in combination with tafamidis, along with statistically significant improvements in multiple measures of disease progression. Vutrisiran’s safety and tolerability were consistent with its established profile. These promising results were published in The New England Journal of Medicine, underscoring its potential as a new treatment option for ATTR-CM patients.
Read Announcement- Drug:
- Vutrisiran (HELIOS-B)
- Announced Date:
- August 7, 2024
- Indication:
- Transthyretin-Mediated (ATTR) Amyloidosis with Cardiomyopathy
Announcement
Alnylam Pharmaceuticals, Inc that the Company will present results from the HELIOS-B Phase 3 study of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy at the European Society of Cardiology (ESC) Congress 2024, taking place in London, UK, on August 30 - September 2, 2024.
AI Summary
Alnylam Pharmaceuticals announced that it will present detailed results from its HELIOS-B Phase 3 study at the European Society of Cardiology (ESC) Congress 2024 in London, UK, scheduled from August 30 to September 2, 2024. The study evaluates vutrisiran in patients with ATTR amyloidosis with cardiomyopathy. In this pivotal trial, the primary endpoint was met, showing a statistically significant reduction in all-cause mortality and recurrent cardiovascular events, while also achieving improvements in secondary endpoints across both the overall and monotherapy populations. The safety profile and tolerability of vutrisiran remained consistent with previous findings. Additionally, the company will host an Investor Webcast on August 30, 2024, to discuss these promising results further. This presentation marks an important milestone in advancing RNAi therapeutics for treating ATTR amyloidosis with cardiomyopathy.
Read Announcement- Drug:
- Vutrisiran (HELIOS-B)
- Announced Date:
- June 24, 2024
- Indication:
- Transthyretin-Mediated (ATTR) Amyloidosis with Cardiomyopathy
Announcement
Alnylam Pharmaceuticals, Inc announced positive topline results from its HELIOS-B Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM).
AI Summary
Alnylam Pharmaceuticals recently announced positive topline results from its HELIOS-B Phase 3 study evaluating vutrisiran for treating ATTR amyloidosis with cardiomyopathy (ATTR-CM). The study met its primary endpoint by significantly reducing the composite of all-cause mortality and recurrent cardiovascular events, with a 28% risk reduction overall and 33% in patients who did not receive tafamidis at baseline. Additionally, vutrisiran showed meaningful improvements in key measures of disease progression such as the six-minute walk test, the Kansas City Cardiomyopathy Questionnaire, and the New York Heart Association Class.
The results also revealed a significant reduction in all-cause mortality over a longer follow-up period. Vutrisiran demonstrated consistent benefits and a favorable safety profile across various patient subgroups. Alnylam plans to file a supplemental New Drug Application using a Priority Review Voucher as it moves forward in making this potential new treatment accessible to patients worldwide.
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Zilebesiran (ALN-AGT) - FDA Regulatory Timeline and Events
Zilebesiran (ALN-AGT) is a drug developed by Alnylam Pharmaceuticals for the following indication: Mild-to-Moderate Hypertension.
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:
- Zilebesiran (ALN-AGT)
- Announced Date:
- April 7, 2024
- Indication:
- Mild-to-Moderate Hypertension
Announcement
Alnylam Pharmaceuticals, Inc. announced positive results from the KARDIA-2 Phase 2 study evaluating the efficacy and safety of a single subcutaneous dose of zilebesiran when added to one of three standard of care antihypertensives including a thiazide-like diuretic (indapamide), calcium channel blocker (amlodipine) or angiotensin receptor blocker (olmesartan).
AI Summary
Alnylam Pharmaceuticals, Inc. announced positive results from the KARDIA-2 Phase 2 study investigating the use of a single subcutaneous dose of zilebesiran when added to standard antihypertensive treatments. The study evaluated zilebesiran in combination with a thiazide-like diuretic (indapamide), a calcium channel blocker (amlodipine), or an angiotensin receptor blocker (olmesartan) in adults with mild-to-moderate hypertension. Results showed that in each cohort, the drug produced clinically significant and additive reductions in blood pressure. Notably, reductions in 24‑hour mean systolic blood pressure reached up to 12.1 mmHg at Month 3, while similar benefits were seen in office systolic blood pressure readings at both Months 3 and 6. The study also demonstrated an encouraging safety and tolerability profile for zilebesiran, suggesting that this RNAi therapeutic could offer sustained blood pressure control with infrequent dosing.
Read Announcement
