NICE recommends vutrisiran for adults with ATTR-CM in final draft guidance - PharmaTimes First long-acting therapy with three-monthly dosing to target root cause of cardiac disease

#AlnylamUK #NICE #vutrisiran #Amvuttra #ATTRCM #NICEfinaldraftguidance #transthyretinamyloidosiswithcardiomyopathy #hereditaryATTR #stage1polyneuropathy #stage2polyneuropathy #NICEfinalguidance #HELIOSBstudy #cardiovascular #stiffheartdisease #AnnaTomlinson #CardiomyopathyUK
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Alnylam's ATTR cardiomyopathy drug backed for NHS use Patients with ATTR-CM in England and Wales will soon have a new treatment option, after NICE backed use of Alnylam's three-month injectable Amvuttra.

#Patients in England and Wales with #cardiomyopathy caused by the disorder #ATTRamyloidosis will soon be able to access treatment with #Alnylam's gene-silencing drug #Amvuttra.

The reason why I'm hesitant about the near-term of #Alnylam (and why they should use their market cap heft while it lasts) is that the list price of #AMVUTTRA is twice that of #ATTRUBY and #VYNDAQEL... not sure how that is sustainable.

Alnylam stock rises following CHMP’s positive opinion on Amvuttra Investing.com -- Shares of Alnylam Pharmaceuticals Inc (NASDAQ:ALNY) climbed 4% following the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommendation to extend the marketing authorization for its drug Amvuttra. The positive opinion, announced on April 25, 2025, supports the use of Amvuttra in treating both wild-type and hereditary transthyretin amyloidosis with cardiomyopathy (ATTR-CM) in adults. Amvuttra, which is already approved for hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN), has now broadened its therapeutic reach, potentially increasing its market presence in the European Union. The CHMP’s endorsement is a significant step towards full marketing authorization for this new indication, which is expected to enhance Alnylam’s product portfolio and revenue streams. The CHMP’s positive opinion is part of a broader set of recommendations for extensions of therapeutic indications for ten medicines already authorized in the EU. The committee’s recognition of Amvuttra’s potential in treating ATTR-CM is anticipated to bolster confidence in Alnylam’s capabilities in addressing rare diseases and expanding treatment options for patients with serious conditions. While Alnylam has not released an official statement regarding the financial impact of the CHMP’s recommendation, investors have responded favorably to the news. The stock’s upward movement reflects optimism about the potential for increased sales and market penetration for Amvuttra in the EU. The market’s reaction underscores the importance of regulatory approvals in shaping pharmaceutical companies’ growth trajectories. As Alnylam awaits the final decision on the marketing authorization for Amvuttra’s new indication, the company’s stock performance will likely continue to be influenced by regulatory developments and the drug’s commercial success in the European market. This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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FDA approves Alnylam’s anti-amyloid RNAi drug for cardiomyopathy - Nature Biotechnology Nature Biotechnology - FDA approves Alnylam’s anti-amyloid RNAi drug for cardiomyopathy

In Brief: US FDA approves the first RNAi drug for treating heart failure caused by amyloidosis. Alnylam gets the nod for #Amvuttra (vutrisiran) to treat transthyretin amyloid cardiomyopathy caused by sporadic or hereditary ATTR, expanding the drug’s label www.nature.com/articles/s41...
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FDA OKs Amvuttra To Treat Heart Conditions - Drugs.com MedNews The U.S. Food and Drug Administration (FDA) has approved a new drug for a serious heart condition that affects thousands of people. The drug, called Amvuttra (v

FDA Approves #Amvuttra for ATTR-CM: A New Weapon Against Rare Heart Disease
Alnylam Pharmaceuticals' drug demonstrated a 28% reduction in heart-related risks over three years, but at a high cost: $476,000/year