An Open-Label Extension Study to Assess the Safety and Efficacy of Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

The purpose of this study is to obtain safety, efficacy, and pharmacodynamic data on the use of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy who continued on extended use of vutrisiran, or switched from patisiran.

Start Date03 Dec 2024

Sponsor / Collaborator

Alnylam Pharmaceuticals, Inc.

NCT04153149 / Active, not recruitingPhase 3

HELIOS-B: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

This study will evaluate the efficacy and safety of vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) compared to placebo in patients with ATTR amyloidosis with cardiomyopathy.

Start Date26 Nov 2019

Sponsor / Collaborator

Alnylam Pharmaceuticals, Inc.

NCT03759379 / Active, not recruitingPhase 3

HELIOS-A: A Phase 3 Global, Randomized, Open-label Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

The purpose of this study is to evaluate the efficacy and safety of vutrisiran (ALN-TTRSC02) in participants with hereditary transthyretin amyloidosis (hATTR amyloidosis). Participants will receive vutrisiran subcutaneous (SC) injection once every 3 months (q3M) or the reference comparator patisiran intravenous (IV) injection once every 3 weeks (q3w) during the 18 month Treatment Period. This study will use the placebo arm of the APOLLO study (NCT01960348) as an external comparator for the primary and most other efficacy endpoints during the 18 Month Treatment Period. Following the 18 Month Treatment Period, all participants will be randomized to receive vutrisiran 50 mg SC injection once every 6 months (q6M) or vutrisiran 25 mg q3M in the Randomized Treatment Extension (RTE) Period. Upon implementation of Amendment 6, participants receiving vutrisiran SC 50 mg q6M will transition to vutrisiran SC 25 mg q3M at their next scheduled dosing.

Start Date14 Feb 2019

Sponsor / Collaborator

Alnylam Pharmaceuticals, Inc.

100 Clinical Results associated with Vutrisiran Sodium

100 Translational Medicine associated with Vutrisiran Sodium

100 Patents (Medical) associated with Vutrisiran Sodium

Literatures (Medical) associated with Vutrisiran Sodium

01 Dec 2024·Giornale Italiano di Cardiologia

[Vutrisiran: a new therapeutic option for cardiac transthyretin amyloidosis].

Article

Author: Panichella, Giorgia ; Aimo, Alberto

01 Nov 2024·JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Outpatient Worsening Heart Failure in Patients with Transthyretin Amyloidosis with Cardiomyopathy in the HELIOS-B Trial

Article

Author: Solomon, Scott D. ; Fontana, Marianna ; Maurer, Mathew S. ; Solomon, Scott D ; Bender, Shaun ; Jay, Patrick Y. ; Eraly, Satish A. ; Aldinc, Emre ; Jay, Patrick Y ; Gillmore, Julian D. ; Eraly, Satish A ; Maurer, Mathew S ; Gillmore, Julian D

BACKGROUND:

Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a fatal disease, caused by misfolded transthyretin depositing as amyloid fibrils in the heart. Because disease progression is common, practical and sensitive methods are needed to monitor patients and optimize treatment decisions. Outpatient worsening heart failure (HF) (oral loop diuretic intensification or initiation) is simple to assess and has been shown to be prognostic of mortality in patients with ATTR-CM.

OBJECTIVES:

We aimed to assess the clinical and prognostic significance of and the effect of vutrisiran treatment on outpatient worsening HF in patients with ATTR-CM from the HELIOS-B trial.

METHODS:

Associations between outpatient worsening HF and a composite of all-cause mortality and recurrent cardiovascular (CV) events (CV hospitalizations and urgent HF visits), all-cause mortality, and other disease progression-related endpoints were evaluated. The impact of vutrisiran over 36 months on outpatient worsening HF and an expanded composite of all-cause mortality, recurrent CV events, and outpatient worsening HF was also assessed.

RESULTS:

Overall, 321 patients (49.1%) had ≥1 outpatient worsening HF, 245 (37.5%) had ≥1 CV event(s), and 120 (18.3%) died; 237 patients (36.2%) had no events. Patients with outpatient worsening HF had an increased risk of all-cause mortality and CV events (hazard ratio [HR]: 2.58; 95% confidence interval [CI]: 2.04-3.27) and all-cause mortality (HR: 2.45; 95% CI: 1.70-3.52), as well as a greater deterioration in 6-minute walk test distance and Kansas City Cardiomyopathy Questionnaire-Overall Summary score, and a greater increase in N-terminal prohormone of B-type natriuretic peptide. In recurrent event analyses over the double-blind period, vutrisiran versus placebo reduced the rate of outpatient worsening HF (relative rate ratio: 0.66; 95% CI: 0.56-0.78). Vutrisiran also reduced the risk of the composite of all-cause mortality, CV events, and outpatient worsening HF versus placebo (HR: 0.69; 95% CI: 0.57-0.83).

CONCLUSIONS:

Outpatient worsening HF was frequent in patients with ATTR-CM in HELIOS-B, and was associated with increased mortality, and reduced by vutrisiran.

01 Nov 2024·Nature Reviews Cardiology

Benefit of vutrisiran in transthyretin amyloidosis with cardiomyopathy.

Article

Author: Lim, Gregory B

Data from the HELIOS-B trial show that RNA interference with vutrisiran reduces the risk of death and recurrent cardiovascular events in patients with transthyretin amyloidosis with cardiomyopathy.

188

News (Medical) associated with Vutrisiran Sodium

25 Nov 2024

·www.businesswire.com

Alnylam Announces U.S. Food and Drug Administration Acceptance of Supplemental New Drug Application for Vutrisiran for the Treatment of ATTR Amyloidosis with Cardiomyopathy

CAMBRIDGE, Mass.--( BUSINESS WIRE )-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today 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). Based on the Company’s use of a Priority Review Voucher, the FDA has set an action date goal of March 23, 2025, under the Prescription Drug User Fee Act (PDUFA). The FDA has informed the Company that it is not planning to hold an advisory committee meeting at this time to review the application. Vutrisiran is the generic name for AMVUTTRA ® , which is currently approved by the FDA for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. By rapidly knocking down both mutant and wild‑type transthyretin (TTR), vutrisiran addresses the underlying cause of transthyretin amyloidosis (ATTR). If approved, vutrisiran would become the first therapeutic approved in the U.S. to treat both the polyneuropathy manifestations of hATTR and cardiomyopathy manifestations of ATTR amyloidosis. “We are pleased that the FDA has accepted our sNDA for vutrisiran for the treatment of ATTR with cardiomyopathy – a steadily progressing, debilitating and ultimately fatal disease,” said Pushkal Garg, M.D., Chief Medical Officer of Alnylam. “In HELIOS-B, treatment with vutrisiran improved cardiovascular outcomes, survival, disease progression and quality of life, as compared to placebo, in a population reflective of today’s patients on substantial background treatment. We look forward to working with the FDA to support their review of the application and bring vutrisiran to patients with ATTR-CM in the U.S. early next year.” The supplemental application to the FDA was based on positive results from HELIOS-B, a randomized, double-blind, placebo-controlled multicenter global Phase 3 study in patients with ATTR-CM. The study demonstrated favorable effects of vutrisiran on outcomes of death and cardiovascular events, functional capacity and quality of life in patients with ATTR-CM. These treatment effects were seen on top of substantial background standard of care treatments. Vutrisiran demonstrated encouraging safety and tolerability consistent with the established profile of the drug. Detailed results from the study were presented at the European Society of Cardiology Congress and simultaneously published in The New England Journal of Medicine on August 30, 2024. AMVUTTRA ® (vutrisiran) U.S. Indication and Important Safety Information Indication AMVUTTRA is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. Important Safety Information Reduced Serum Vitamin A Levels and Recommended Supplementation AMVUTTRA treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking AMVUTTRA. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with AMVUTTRA, as serum vitamin A levels do not reflect the total vitamin A in the body. Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness). Adverse Reactions The most common adverse reactions that occurred in patients treated with AMVUTTRA were pain in extremity (15%), arthralgia (11%), dyspnea (7%), and vitamin A decreased (7%). For additional information about AMVUTTRA, please see the full U.S. Prescribing Information . About AMVUTTRA ® (vutrisiran) AMVUTTRA ® (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of mutant and wild-type transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. Administered quarterly via subcutaneous injection, AMVUTTRA is approved and marketed in more than 15 countries for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. Vutrisiran is also in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM), which encompasses both wild-type and hereditary forms of the disease. For more information about AMVUTTRA, including the full U.S. Prescribing Information , visit AMVUTTRA.com . About ATTR Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy, or both manifestations of disease. There are two different forms of ATTR – hereditary ATTR (hATTR), which is caused by a TTR gene variant and affects approximately 50,000 people worldwide, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant and impacts an estimated 200,000 – 300,000 people worldwide. About RNAi RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors that encode for disease-causing or disease pathway proteins – thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases. About Alnylam Pharmaceuticals Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam’s commercial RNAi therapeutic products are ONPATTRO® (patisiran), AMVUTTRA® (vutrisiran), GIVLAARI® (givosiran), OXLUMO® (lumasiran), and Leqvio® (inclisiran), which is being developed and commercialized by Alnylam’s partner, Novartis. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its “ Alnylam P 5 x25 ” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on X (formerly Twitter) at @Alnylam , or on LinkedIn , Facebook , or Instagram . Alnylam Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam’s expectations, beliefs, goals, plans or prospects including, without limitation, Alnylam’s expectations regarding the safety and efficacy of vutrisiran for the treatment of ATTR amyloidosis with cardiomyopathy, including the safety, tolerability and favorable effects of vutrisiran on outcomes of death and cardiovascular events, functional capacity and quality of life in patients with ATTR-CM; whether the FDA will convene an advisory committee in connection with the FDA’s review of Alnylam’s supplemental application for approval of vutrisiran for the treatment of patients with ATTR-CM; the potential for vutrisiran to obtain FDA approval for the treatment of patients with ATTR-CM, and the anticipated timing of such FDA approval; and the potential for Alnylam to achieve its Alnylam P 5 x25 vision of becoming a leading biopharma company should be considered forward-looking statements. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to: Alnylam’s ability to successfully execute on its “ Alnylam P 5 x25 ” strategy; Alnylam’s ability to successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam’s product candidates, including vutrisiran; actions or advice of regulatory agencies and Alnylam’s ability to obtain regulatory approval for its product candidates, including vutrisiran, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam’s approved products globally; and any delays, interruptions or failures in the manufacture and supply of Alnylam’s product candidates or its marketed products; as well as those risks more fully discussed in the “Risk Factors” filed with Alnylam’s 2023 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam’s subsequent Quarterly Reports on Form 10-Q and in its other SEC filings. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

Clinical ResultDrug ApprovalPhase 3NDAPriority Review

25 Nov 2024

·firstwordpharma.com

Alnylam cashes in priority review voucher to catch up to BridgeBio in ATTR-CM race

With last week's FDA approval of Attruby (acoramidis), BridgeBio may reach transthyretin amyloid cardiomyopathy (ATTR-CM) patients first, but Alnylam isn't far behind. The company used a priority review voucher as part of its regulatory submission for RNAi therapeutic vutrisiran, which the US regulator accepted on Monday and granted a PDUFA date of March 23, 2025. BridgeBio and Alnylam are both vying for a piece of the ATTR-CM market, which Pfizer currently dominates. The pharma's tafamidis — marketed as Vyndamax and Vyndaqel — became the first drug approved for the rare heart disease in 2019, and last year brought in a total of $3.3 billion in global sales. While BridgeBio's Attruby, an oral TTR stabiliser, has the same mechanism of action as tafamidis, Alnylam's asset is a wholly separate modality. Vutrisiran is designed to deliver rapid knockdown of mutant and wild-type transthyretin (TTR), the disease's underlying cause. The RNAi therapeutic was approved in 2022 as Amvuttra to treat polyneuropathy in adults with hereditary transthyretin-mediated (hATTR) amyloidosis.ATTR anticipationCardiologists have been excited about vutrisiran's potential in ATTR-CM since Alnylam top-lined data from the Phase III HELIOS-B trial in June. In the overall study population, vutrisiran reduced all-cause mortality and recurrent cardiovascular events by 28% compared to placebo (see – Physician Views Results: Cardiologist feedback suggests Alnylam well placed to take on Pfizer in ATTR-CM field).Further results from HELIOS-B shared in August at the European Society of Cardiology (ESC) congress demonstrated that in the overall study population, vutrisiran reduced the relative risk of death by 31% through month 36, and by 36% after up to 42 months. However, patients not receiving tafamidis at the trial's start saw a non-significant risk reduction of 30% at 36 months. After the data drop, FirstWord spoke with Sarju Ganatra, director of the Cardio-Oncology Programme and co-director of the South Asian Cardio-Metabolic Programme at Lahey Hospital and Medical Center, who said the monotherapy arm showed a "lacklustre performance on the mortality benefit." For the full interview, see KOL Views Q&A: HELIOS-B cements pecking order for Alnylam’s Amvuttra, Pfizer’s Vyndamax in ATTR-CM.A pair of physician polls conducted after the ESC presentation showed that vutrisiran is on track to become an important new treatment option for ATTR-CM, though the majority of cardiologists noted that it was likely to be used most frequently alongside other treatment options (see Physician Views Poll Results: Amvuttra another compelling treatment option for ATTR-CM, say majority of cardiologists and Physician Views Results: Cardiologists have much to ponder with ATTR-CM treatment options set to expand).Meanwhile, Alnylam also has next-generation siRNA therapeutic nucresiran in early-stage testing for ATTR. The drug recently demonstrated rapid and durable reduction of serum TTR in healthy participants.

Drug ApprovalClinical ResultPhase 3Priority ReviewAHA

25 Nov 2024

·medcitynews.com

FDA Nod for BridgeBio Brings New Competition to Blockbuster Pfizer Cardio Drug - MedCity News

A BridgeBio Pharma drug developed for an increasingly prevalent cardiovascular condition has won FDA approval, marking a comeback for a company and a molecule with a turbulent history. But BridgeBio’s challenges aren’t over. The biotech’s new product must now compete against a blockbuster Pfizer medication already well established as the standard of care and fend off new competition on the way. The late Friday approval for the drug, acoramidis, covers the treatment of adults with cardiomyopathy caused by transthyretin-mediated amyloidosis, or ATTR. The disease stems from a genetic mutation though it can also develop as a consequence of aging. The FDA approval covers both types. BridgeBio’s twice-daily pill will be marketed under the brand name Attruby. In ATTR, abnormal versions of a liver protein called transthyretin lead to a buildup of amyloid protein in tissues and organs. When this buildup affects the heart, it can lead to cardiomyopathy, making it harder for the organ to pump blood. Attruby is a small molecule designed to bind to the abnormal TTR, stabilizing the protein and preventing it from leading to the amyloid buildup characterized by the disease. Pfizer’s ATTR cardiomyopathy drug, tafamidis, approved in 2019 and marketed as the four-times daily capsule Vyndaqel and the once-daily capsule Vyndamax, is also a TTR stabilizer. But Palo Alto, California-based BridgeBio set out to show its drug is the better stabilizer. That effort was almost derailed. Health IT Reducing Clinical and Staff Burnout with AI Automation As technology advances, AI-powered tools will increasingly reduce the administrative burdens on healthcare providers. By Dr. Michael Blackman, Chief Medical Officer at Greenway Health Nearly three years ago, BridgeBio reported preliminary data showing its drug failed to beat a placebo in the first part of a Phase 3 clinical trial, which assessed patients after 12 months. The company attributed the failure to a high placebo response. The disappointing results sank shares of the biotech by more than 70%. But the study’s design included a second part that would assess patients after 30 months of treatment. BridgeBio opted to continue the study, hoping longer treatment would lead to better outcomes. FDA approval of Attruby is based on the 30-month data from the Phase 3 study, which enrolled 632 patients with ATTR cardiomyopathy. Results showed the drug met the main goal of showing statistically significant improvement on a composite endpoint comprised of four measures of the disease. The results were published in the New England Journal of Medicine early this year. Speaking during a Friday evening webcast, BridgeBio CEO Neil Kumar drew distinctions between Attruby and Pfizer’s product. “This is the only oral stabilizer with the verbiage ‘near-complete stabilization’ in the label,” he said. “Almost all of benefits described downstream in the label stem from this near-complete stabilization. As early as three months, the earliest time point that we know of, this medicine begins to take action against a composite endpoint of cardiovascular hospitalization and death.” BridgeBio estimates 500,000 patients in the U.S. and European Union have ATTR cardiomyopathy, which represents a market opportunity of $15 billion to $20 billion. Right now, that market is dominated by Pfizer, which reported $3.9 billion in tafamidis sales in the first nine months of 2024, a more than 65% increase compared to the same period in 2023. But this market could soon be split among several products. Consumer / Employer Health Executives on Digital Transformation in Healthcare Hear executives from Quantum Health, Surescripts, EY, Clinical Architecture and Personify Health share their views on digital transformation in healthcare. By MedCity News Alnylam Pharmaceuticals develops drugs that silence genes, preventing them from causing disease-causing proteins. The biotech already markets Amvuttra for treating polyneuropathy caused by ATTR. But Alnylam has also tested Amvuttra, administered as a subcutaneous injection every three months, as a treatment for ATTR cardiomyopathy. On Monday, Alnylam announced the FDA accepted the company’s supplemental new drug application in this indication, setting a March 23, 2025 target date for a regulatory decision. Meanwhile, partners AstraZeneca and Ionis Pharmaceuticals market antisense oligonucleotide drug eplontersen, brand name Wainua, for treating polyneuropathy caused by ATTR. A Phase 3 trial is underway testing the drug in ATTR cardiomyopathy. In a note sent to investors, Leerink Partners analyst Mani Foroohar said Attruby’s label was the best-case scenario as the indication statement includes cardiovascular mortality and hospitalization, consistent with Pfizer’s product. Furthermore, Attruby can stake the claim of being the first and only product with a label specifying near-complete stabilization of TTR. That said, cracking Pfizer’s established market presence will be difficult. In results of a Leerink survey released in September, the Pfizer drug was physicians’ treatment of choice for cardiomyopathy caused by ATTR, given long their long experience with the product. There was an uptick in preference for gene silencing therapies, but mainly for treating polyneuropathy caused by the disease, the survey showed. Many physicians stated they would switch to another product only if a patient’s condition is not well controlled by the Pfizer drug. William Blair analyst Myles Minter said language in Attruby’s label stating the drug’s ability to reduce cardiovascular death is “a clear win for BridgeBio.” But Minter noted the absence on the label of any standalone all-cause mortality data, which is different than the Pfizer product’s label. Attruby did not lead to a statistically significant benefit on all-cause mortality compared to a placebo in its pivotal study. Minter said clinicians the firm spoke with at recent cardiovascular conferences continue to suggest the importance of not only the magnitude of cardiovascular event risk reduction, but also the timing of the onset of the drug’s effect given that cardiomyopathy can progress rapidly. He added that TTR-silencing drugs are best positioned for market differentiation. “We continue to view a reduction in all-cause mortality on label as a necessary distinction to gain major market share in ATTR-CM alongside Pfizer’s tafamidis,” Minter said. “Given the lack of all-cause mortality data included in the Attruby label, we believe Pfizer could start marketing more specifically around mortality benefit claims.” BridgeBio set Attruby’s wholesale price at $18,759.12 for a 28-day supply, which works out to about $244,500 annually. That’s a slight discount to Pfizer’s product, priced at $267,987 annually. But it’s still well above the $13,600 to $39,000 cost-effectiveness price range calculated by the Institute for Clinical and Economic Review, a non-profit organization that researches the value of drugs in order to determine what would be a fair price. Kumar said all patients who participated in Attruby’s Phase 3 study will continue to receive the drug for free for the rest of their lives. Attruby is still under review in Europe with a regulatory decision expected in 2025. Under a deal struck earlier this year, Bayer has exclusive rights to market the drug in Europe for ATTR cardiomyopathy. Bayer shelled out $310 million in upfront and near-term milestone payments; additional sales-based milestones were not disclosed. BridgeBio will also earn royalties from Bayer’s sales of the product. Image: Magicmine, Getty Images

Phase 3Clinical ResultDrug Approval

100 Deals associated with Vutrisiran Sodium

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KEGG	Wiki	ATC	Drug Bank
D11916 D11917	-	-	DB16699

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Transthyretin-mediated amyloidosis	AU	Medison Pharma Australia Pty Ltd.	21 Jun 2024
Amyloidosis, Hereditary, Transthyretin-Related	US	Alnylam Pharmaceuticals, Inc.	13 Jun 2022

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Transthyretin Amyloid Cardiomyopathy	NDA/BLA	US	Alnylam Pharmaceuticals, Inc.	09 Oct 2024
Stargardt Disease	Phase 2	US	Alnylam Pharmaceuticals, Inc.	08 Oct 2022

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04153149 (HELIOS-B, BusinessWire) Manual	Phase 3	Transthyretin Amyloid Cardiomyopathy N-terminal pro-B-type Natriuretic peptide \| Cardiac Troponin I	655	Vutrisiran 25mg	rtjyqyziet(kxjcldxjqn) = cbaqaleayy drxnbgydwa (mxvhyiktiy ) View more	Positive	29 Sep 2024
NCT04153149 (HELIOS-B, Pubmed \| N Engl J Med) Manual	Phase 3	Transthyretin Amyloid Cardiomyopathy	655	Vutrisiran 25 mg	wxbzbmrbiw(hveuyoiozp): HR = 0.72 (95% CI, 0.56 - 0.93), P-Value = 0.01 View more	Positive	30 Aug 2024
				Placebo
NCT04153149 (HELIOS-B, Company_Website) Manual	Phase 3	Transthyretin Amyloid Cardiomyopathy	655	Vutrisiran (overall population)	nmnfdkzkoh(imzoxdeaxd) = Rates of adverse events (AEs), serious AEs and AEs leading to study drug discontinuation were similar between the vutrisiran and placebo arms. No AEs were seen ≥3% more frequently in the vutrisiran arm compared to the placebo arm. iwkkdjegre (qflbnpdfvr )	Positive	24 Jun 2024
				Vutrisiran (not receiving tafamidis at baseline)
NCT03759379 (HELIOS-A, Pubmed)	Phase 3	Amyloid Neuropathies, Familial	164	Vutrisiran	qmboblttnr(hqqrotjylt) = jzjikhpyhr hvpzffhany (ppxsnmcdhw )	-	31 Jul 2023
NCT01960348 \| NCT03759379 (APOLLO, ANA2022)	Phase 3	Polyneuropathies \| Amyloidosis NfL	-	Patisiran	qrxlkecumq(gywptxwyis) = kfaanwxwrv vowhmgwjkt (sddghgmudr )	Positive	14 Sep 2022
				Vutrisiran	qrxlkecumq(gywptxwyis) = ecjtobusdg vowhmgwjkt (sddghgmudr )
NCT03759379 (HELIOS-A, Corporate Publications) Manual	Phase 3	Cardiomyopathies	199	vutrisiran	shfvtasmbh(dkephzweli) = mrximicyhn wtzrjcrums (uteftjoxqu )	Positive	23 May 2022
				Placebo	shfvtasmbh(dkephzweli) = ogzrtvmefv wtzrjcrums (uteftjoxqu )
NCT03759379 (HELIOS-A, Corporate Publications) Manual	Phase 3	Amyloid Neuropathies	199	Vutrisiran	rwnrjnrzbq(paydoothkg) = emnoufxfnx efryuqfwww (aqoxmbzifo ) View more	Positive	21 Jan 2022
				Placebo	rwnrjnrzbq(paydoothkg) = wqanjoehvg efryuqfwww (aqoxmbzifo ) View more

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