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Alnylam's Amvuttra turns in 'grand slam' showing in ATTR-CM, but leaves a window open for rivals

Fierce Pharma

a “grand slam” with data that position its RNA silencer Amvuttra (vutrisiran) to potentially be | At the European Society of Cardiology conference in London, Alnylam presented positive data from the phase 3 HELIOS-B trial which assessed RNA silencer Amvuttra (vutrisiran) as a treatment for transthyretin amyloid cardiomyopathy (ATTR-CM).

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LIB Therapeutics’ Cholesterol Lowering Med Lerodalcibep Receives FDA BLA Acceptance

XTalks

Current PCSK9 inhibitors on the market include Regenerons monoclonal antibody Praluent (alirocumab), Amgens Repatha (evolocumab) and Novartiss Leqvio (inclisiran), which is a small RNA interference (siRNA) therapy designed to lower LDL-C. Over 2,400 patients have continued in the 72-week open-label extension trial.

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Vutrisiran Reduces Heart Risks for ATTR-CM Patients in Alnylam’s HELIOS-B Trial

XTalks

Alnylam Pharmaceuticals announced promising results from its HELIOS-B Phase III clinical trial evaluating vutrisiran, an investigational RNA interference (RNAi) therapeutic for treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM). As of March 2024, the FDA has approved six small interfering RNA (siRNA) therapies.

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Novartis tries to rescue stalled inclisiran filing with FDA

pharmaphorum

The small interfering RNA (siRNA) therapy has already been approved in Europe as Leqvio , and the FDA’s rejection was not caused by any issues with its efficacy or safety, according to Novartis. Novartis added inclisiran to its pipeline after buying The Medicines Company, which had licensed the drug from Alnylam, for $9.7

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Mission to MAARS: Long non-coding RNA may play a key role in cardiovascular disease

Scienmag

Mouse model helped team find MAARS lncRNA, which may contribute to atherosclerotic plaque buildup Atherosclerosis is marked by the buildup of inflammatory cells which narrow arteries to the point of chest pain and muscle weakness. Severe cases result in lesions and internal ruptures of arteries or even thrombosis in coronary arteries.

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Cellular pathway of genetic heart disease similar to neurodegenerative disease

Scienmag

This landmark discovery found a correlation between the clumping of RNA-binding proteins long linked to neurodegenerative disease ? . — Research on a genetic heart disease has uncovered a new and unexpected mechanism for heart failure. and the aggregates of protein found in the heart tissue of patients with RBM20 dilated cardiomyopathy.

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FDA rejects Novartis’ cholesterol drug after factory inspection issue

pharmaphorum

Inclisiran is a small interfering RNA (siRNA) therapy that works by inhibiting PCSK9 – the same target as Amgen’s Repatha (evolocumab) and Sanofi/Regeneron’s Repatha (alirocumab) – but is dosed only twice a year rather than every month.

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