Skip to content
ContentLora

    Tip: press / anywhere to search.

    organization

    Alnylam Pharmaceuticals

    Also known as Alnylam, ALNY

    Alnylam Pharmaceuticals is the company behind Onpattro, the first approved RNA interference (RNAi) drug; it had six approved medicines as of July 2026.[1][2] After Amvuttra's 2025 approval for ATTR cardiomyopathy, its quarterly sales passed $1 billion in mid-2026.[3][4]

    Editor reviewedStrict sourcingUpdated mRNA and next-gen vaccinesHealth and medicineLife sciences
    Key facts

    Alnylam Pharmaceuticals develops medicines based on RNA interference (RNAi). RNAi uses small interfering RNAs to silence the messenger RNA for a disease-causing protein.[1] It matters because it brought the first RNAi drug to market. In 2026 one of its RNAi drugs passed $1 billion in quarterly sales.[3][4]

    From Onpattro to Amvuttra

    On 10 August 2018 the FDA approved Onpattro (patisiran), the first RNAi therapeutic. It treats polyneuropathy caused by hereditary transthyretin (ATTR) amyloidosis by silencing the mRNA that encodes transthyretin.[1] Onpattro is a lipid formulation infused every three weeks.[5] Its successor, Amvuttra (vutrisiran), is a subcutaneous injection given quarterly. Amvuttra was first approved in 2022 for hereditary ATTR polyneuropathy, based on the HELIOS-A trial.[6] On 20 March 2025 the FDA approved Amvuttra for ATTR cardiomyopathy based on the phase 3 HELIOS-B trial.[3]

    The HELIOS-B result

    The cardiomyopathy approval moved RNAi from a rare inherited disease into a much more common heart condition.[3] Alnylam reported that HELIOS-B met all 10 of its prespecified primary and secondary endpoints. In the overall population, Amvuttra cut the risk of death from any cause or recurrent cardiovascular events by 28% compared with placebo, over a double-blind period of up to 36 months. These are company-reported figures.[7] In the earlier HELIOS-A trial, the most common adverse reactions were pain in the arms or legs (15%), joint pain (11%), shortness of breath (7%) and lower vitamin A levels (7%).[6] Many newer silencing drugs attach GalNAc, a sugar that drives uptake into liver cells, instead of using an infused nanoparticle.[8] See how RNA silencing drugs work.

    How its drugs work

    siRNA drugs load into the RNA-induced silencing complex (RISC). Its AGO2 enzyme then cleaves the matching mRNA or blocks its translation, so less of the target protein is made.[9] Onpattro used a lipid formulation, and lipid nanoparticles naturally accumulate in the liver.[5][10] GalNAc conjugates bind the asialoglycoprotein receptor on hepatocytes and give efficient uptake without a nanoparticle.[8] That is why transthyretin and other proteins made in the liver have been the field’s first targets.[1][8]

    Commercial scale

    In the second quarter of 2026 Alnylam reported net product revenues of about $1.17 billion, with Amvuttra contributing about $1.01 billion. Amvuttra therefore accounted for most of the quarter’s product revenue.[4] In the same report it lowered full-year 2026 TTR revenue guidance to $4.2-4.5 billion, from $4.4-4.7 billion.[11] As of July 2026 it had six approved medicines.[2]

    Pipeline

    Alnylam is running ZENITH, a phase 3 trial of zilebesiran. It is also running phase 2 trials of mivelsiran in Alzheimer’s disease, including Alzheimer’s associated with Down syndrome.[12] Most current oligonucleotide and nanoparticle delivery is concentrated in the liver.[10][8]

    Place in the field

    Alnylam sits on the silencing side of RNA medicine. mRNA companies such as Moderna sit on the expression side, adding instructions rather than removing them.[13] Antisense drugmakers form a parallel class. Their single-stranded drugs often work through the enzyme RNase H1 rather than RISC.[14] A February 2026 review counted sixteen antisense drugs approved by the FDA and the European Medicines Agency, including olezarsen for familial chylomicronemia syndrome in 2024.[15][16]

    This page describes company activities and regulatory status. It is not medical advice.

    Questions readers ask

    What was Alnylam's first approved drug?

    Onpattro (patisiran), approved by the FDA on 10 August 2018 as the first RNAi therapeutic, for hereditary ATTR amyloidosis polyneuropathy.[1]

    What is Amvuttra approved for?

    In March 2025 the FDA approved it for the cardiomyopathy of transthyretin amyloidosis (ATTR-CM), based on the phase 3 HELIOS-B trial. It is injected under the skin quarterly.[3]

    Did Alnylam change its 2026 sales guidance?

    Yes. In July 2026 it lowered its full-year TTR revenue guidance to $4.2-4.5 billion from $4.4-4.7 billion.[11]

    Sources

    Each numbered claim is a statement we checked against the sources listed with it. Status shows how well established it is.

    1. [1]

      On 10 August 2018 the FDA approved Alnylam's Onpattro (patisiran), the first approved RNAi therapeutic, which silences the mRNA encoding transthyretin to treat hereditary ATTR amyloidosis polyneuropathy. confirmedas of 2018-08-10

    2. [2]

      As of July 2026 Alnylam had six approved medicines. confirmedas of 2026-07-30

    3. [3]

      On 20 March 2025 the FDA approved Alnylam's RNAi drug Amvuttra (vutrisiran) for the cardiomyopathy of transthyretin amyloidosis (ATTR-CM), based on the phase 3 HELIOS-B trial, with quarterly subcutaneous dosing. confirmedas of 2025-03-20

    4. [4]

      Alnylam reported second-quarter 2026 net product revenues of about $1.17 billion, of which Amvuttra contributed about $1.01 billion. confirmedas of 2026-07-30

    5. [5]

      Onpattro is a lipid formulation given by intravenous infusion once every three weeks. confirmedas of 2018-08-10

    6. [6]

      Amvuttra was first approved by the FDA in 2022 for polyneuropathy of hereditary ATTR amyloidosis, based on the HELIOS-A trial; the most common adverse reactions in that trial were pain in extremity (15%), joint pain (11%), shortness of breath (7%) and decreased vitamin A (7%). confirmedas of 2025-03-20

    7. [7]

      Alnylam reported (self-reported) that in the phase 3 HELIOS-B trial Amvuttra met all 10 prespecified primary and secondary endpoints and reduced the risk of all-cause mortality and recurrent cardiovascular events by 28% versus placebo over a double-blind period of up to 36 months. confirmedas of 2025-03-20

    8. [8]

      GalNAc is a sugar attached to oligonucleotides that binds the asialoglycoprotein receptor on liver cells and drives efficient uptake into hepatocytes. confirmedas of 2026-02-01

    9. [9]

      Oligonucleotides can also act through the RNA-induced silencing complex (RISC), whose AGO2 enzyme cleaves or blocks translation of the matching mRNA. confirmedas of 2026-02-01

    10. [10]

      Lipid nanoparticles in the blood adsorb apolipoprotein E and therefore accumulate preferentially in the liver, which limits delivery to other organs. confirmedas of 2023-03-01

    11. [11]

      In July 2026 Alnylam lowered its full-year 2026 TTR net product revenue guidance from $4.4-4.7 billion to $4.2-4.5 billion. confirmedas of 2026-07-30

    12. [12]

      As of July 2026 Alnylam was running the ZENITH phase 3 trial of zilebesiran and phase 2 trials of mivelsiran in Alzheimer's disease. confirmedas of 2026-07-30

    13. [13]

      Two major types of RNA are studied as vaccines, non-replicating mRNA and virally derived self-amplifying RNA. confirmedas of 2018-01-12

    14. [14]

      Many ASOs work by forming a DNA-RNA duplex with their target, which the enzyme RNase H1 recognises and cleaves, reducing production of the encoded protein. confirmedas of 2026-02-01

    15. [15]

      A February 2026 review counted sixteen antisense oligonucleotide drugs approved by the US FDA and the European Medicines Agency. confirmedas of 2026-02-01

    16. [16]

      The antisense drug olezarsen was approved in 2024 to treat severe hypertriglyceridemia in people with familial chylomicronemia syndrome. confirmedas of 2026-02-01

    Revision history (2)
    1. Page created.
    2. Added Amvuttra's 2022 first approval, HELIOS-B results and safety profile; corrected the antisense approval count.

    Created Oct 10, 2026. Last reviewed by an editor on Oct 10, 2026. Next scheduled review: Jan 10, 2027.

    Cite this page

    "Alnylam Pharmaceuticals." ContentLora, updated Oct 10, 2026. https://contentlora.com/wiki/alnylam-pharmaceuticals

    Spotted an error? Suggest a correction or emailcorrections@contentlora.com.

    Keep exploring

    Connections
    Moderna Peer in RNA medicines
    ☰ Contents