Alnylam Pharmaceuticals is a biopharmaceutical company focused on advancing innovative therapies using RNA interference (RNAi) technology to treat a range of genetic diseases
The company is dedicated to developing medicines that can selectively silence disease-causing genes, thereby addressing the underlying causes of conditions such as hereditary transthyretin-mediated amyloidosis and other rare disorders. With a strong commitment to research and development, Alnylam aims to deliver transformative treatments that improve patient outcomes and enhance quality of life for those affected by severe or life-threatening diseases.
FDA approves Sanofi's Qfitlia, the first antithrombin-lowering therapy for hemophilia A or B, reducing bleeding episodes with as few as six injections per year.
Precision medicine was proved in the ATTR-CM field. We think acoramidis is a more potent TTR stabilizer and that it will attract first-line patients. Tafamidis, acoramidis and vutrisiran will share the ATTR-CM market in the next 3 years.
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today 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. Data were presented at the American College of Cardiology’s Annual Scientific Session (ACC.25) held in Chicago, Illinois. These data build upon the body of clinical evidence which supported the recent U.S. Food and Drug Administration (FDA) approval of AMVUTTRA® (vutrisiran) for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits. AMVUTTRA is an RNAi therapeutic that works upstream to deliver rapid knockdown of transthyretin, addressing the disease at its source, with four subcutaneous doses per year.
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, 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. Qfitlia is indicated in the U.S. for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or B, with or without factor VIII or IX inhibitors (neutralizing antibodies). Alnylam scientists discovered Qfitlia, published the first clinical data in the New England Journal of Medicine in 2017 showing reduced bleeding rates in hemophilia patients, and initiated the Phase 3 development program. In 2014, Sanofi obtained global rights to co-develop and co-commercialize Qfitlia under a license and collaboration agreement. The agreement was amended in 2018, with Sanofi obtaining global development and commercialization rights to Qfitlia, and Alnylam becoming eligible to receive tiered royalties of 15 to 30 percent on global net sales.
Alnylam's Amvuttra wins FDA approval for ATTR-CM, expanding its market reach. JP Morgan upgrades the stock, projecting strong sales and a multi-billion opportunity.
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Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today 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. The approval expands the indication for AMVUTTRA, which now becomes the first and only therapeutic approved by the FDA for the treatment of ATTR-CM and the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that management will present a company overview at the Stifel 2025 Virtual CNS Forum on Tuesday, March 18, 2025 at 3:00 pm ET.
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the retirement of Dr. Phillip A. Sharp, Ph.D., from the Company’s Board of Directors, effective as of May 8, 2025. Dr. Sharp has served as a key advisor to Alnylam since he co-founded the Company in 2002. Dr. Sharp will remain a member of the Alnylam Scientific Advisory Board.
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, is hosting an R&D Day today in New York City and will also webcast the event. The Company plans to showcase its R&D progress and platform innovation, including updates on multiple near- and mid-stage potentially transformative therapies that represent blockbuster opportunities as its pipeline rapidly expands across multiple therapeutic areas.
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the fourth quarter and full year ended December 31, 2024 and reviewed recent business highlights.
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that it will report financial results for the fourth quarter and year ending December 31, 2024 on Thursday, February 13, 2025, before the U.S. financial markets open.
