Does Alnylam work only on rare diseases?

No. The 4 medicines Alnylam sells treat rare diseases, but its pipeline also reaches common conditions. Rare disease medicines: Amvuttra and Onpattro for ATTR amyloidosis, Givlaari for acute hepatic porphyria and Oxlumo for primary hyperoxaluria type 1. Common conditions: zilebesiran for high blood pressure is in a Phase 3 trial (NCT07181109), and the pipeline lists programs in obesity, type 2 diabetes and Alzheimer's disease. Partner medicines: Novartis sells Leqvio for high cholesterol and Sanofi sells Qfitlia for hemophilia, both built on Alnylam's RNAi work. Source: Alnylam pipeline, checked November 9, 2026.

What rare diseases does Alnylam Pharmaceuticals focus on?

Alnylam Pharmaceuticals has active programs across 9 rare diseases: Acute Hepatic Porphyria, ATTR Amyloidosis (Transthyretin Amyloidosis), Hemophilia A, Hemophilia B, Hereditary Hemorrhagic Telangiectasia, Huntington Disease, Paroxysmal Nocturnal Hemoglobinuria, Primary Hyperoxaluria, Von Willebrand Disease. These span several therapeutic areas. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.

How can I find Alnylam Pharmaceuticals clinical trials for rare diseases?

The clinical trials section on this page shows active and recruiting rare disease trials sponsored by Alnylam Pharmaceuticals, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.

How do I enroll in an Alnylam Pharmaceuticals clinical trial?

To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.

What FDA-approved rare disease drugs does Alnylam Pharmaceuticals have?

Alnylam Pharmaceuticals has 4 FDA-approved drugs with a rare disease use that we track, as of November 2026. Onpattro (patisiran), approved 2018: ATTR Amyloidosis. Amvuttra (vutrisiran), approved 2022: ATTR Amyloidosis, Wild-Type ATTR Cardiac Amyloidosis. Oxlumo (lumasiran), approved 2020: Primary Hyperoxaluria. Givlaari (givosiran), approved 2019: Acute Hepatic Porphyria. Still in development: ALN-6400 (Phase 2). Source: the FDA's openFDA label database, matched to the rare diseases on Trial Friend; the list updates automatically and can include older products.

Does Alnylam Pharmaceuticals offer copay help or free medicine for its rare disease drugs?

Yes, for 4 of its FDA-approved rare disease medicines that we track, each checked on Alnylam Pharmaceuticals' own website. Onpattro: Alnylam Assist, 1-833-256-2748. Offers copay help with private insurance, a free medicine program, a short-term supply while insurance is decided and insurance help. Checked September 24, 2026. Amvuttra: Alnylam Assist, 1-833-256-2748. Offers copay help with private insurance, a free medicine program, a short-term supply while insurance is decided and insurance help. Checked September 24, 2026. Oxlumo: Alnylam Assist, 1-833-256-2748. Offers copay help with private insurance, a free medicine program and insurance help. Checked October 24, 2026. Givlaari: Alnylam Assist, 1-833-256-2748. Offers copay help with private insurance, a free medicine program, a short-term supply while insurance is decided and insurance help. Checked October 18, 2026. Medicare and Medicaid: drugmaker copay cards are limited to private insurance, so people with government coverage usually look to a free medicine program or a charity fund, both on the help paying page. Eligibility is decided by each program, so call the number listed before a prescription is filled.

Alnylam Pharmaceuticals

Rare disease drugmaker · based in Cambridge, United States · founded 2002 · ticker ALNY · alnylam.com

Alnylam Pharmaceuticals works on 9 rare diseases we track, including Acute Hepatic Porphyria, ATTR Amyloidosis (Transthyretin Amyloidosis) and 7 more. It has 13 active clinical trials, and 9 are looking for volunteers now. It also has 4 FDA-approved rare disease drugs.

Alnylam Pharmaceuticals is one of the longest bets in modern biotech. The company was founded in Cambridge, Massachusetts in 2002 to commercialize a then-new technology called RNA interference (RNAi), a gene-silencing mechanism that would later win its discoverers the Nobel Prize in Physiology or Medicine in 2006. RNAi works by using small pieces of RNA to silence specific disease-causing genes before the body can make harmful proteins from them.

Alnylam Pharmaceuticals pipeline

Each row lists the diseases Alnylam Pharmaceuticals is studying at that stage, from the ones closest to approval down to the earliest work.

Approved

Drugs the FDA has approved.

Amvuttra (vutrisiran) · Givlaari (givosiran sodium) · Onpattro (patisiran) · Oxlumo (lumasiran)

Phase 3

Large studies, usually the last step before a company asks the FDA for approval.

ATTR Amyloidosis (Transthyretin Amyloidosis) 2 trials recruiting · Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy recruiting

Phase 2

Mid-size studies that check whether a drug works and at what dose.

Von Willebrand Disease recruiting · Hereditary Hemorrhagic Telangiectasia

Phase 1

First studies in people, mostly checking safety.

Huntington Disease recruiting

Other studies

Studies with no phase listed, such as registries and natural history studies.

ATTR Amyloidosis (Transthyretin Amyloidosis) 4 trials recruiting · Acute Hepatic Porphyria recruiting · Hereditary Amyloidosis, Transthyretin-Related · Primary Hyperoxaluria

Alnylam Pharmaceuticals clinical trials

These are Alnylam Pharmaceuticals trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.

9 trials looking for volunteers now

Looking for volunteersPhase 2Recently updatedNCT07575308

HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB

Tests ALN-6400

For Von Willebrand Disease (VWD) and Heavy Menstrual Bleeding (HMB)

The purpose of this study is to evaluate the safety, tolerability, efficacy, and pharmacodynamics (PD) of multiple doses of ALN-6400 in adult and adolescent patients with VWD and HMB

Ages 16 Years - 45 Years29 locations

View full study

Looking for volunteersPhase 3Recently updatedNCT07052903

TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

Tests Nucresiran, Sterile Normal Saline (0.9% NaCl)

For Transthyretin Amyloidosis With Cardiomyopathy

The purpose of this study is to: * Evaluate the efficacy of nucresiran compared to placebo on reducing all-cause mortality and cardiovascular (CV) events * Evaluate the efficacy of nucresiran compared to placebo on additional assessments of CV events and/or death * Evaluate the efficacy of nucresiran compared to placebo on patient-reported health status and health-related quality of life

Ages 18 Years - 85 Years266 locations

View full study

Looking for volunteersPhase 1Recently updatedNCT06585449

A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease

Tests ALN-HTT02, Placebo

For Huntington's Disease

The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single or repeat doses of ALN-HTT02.

Ages 25 Years - 70 Years19 locations

View full study

Looking for volunteersRecently updatedNCT04561518

ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)

For Transthyretin-Mediated Amyloidosis and ATTR Amyloidosis

The purpose of this study is to: * Describe epidemiological and clinical characteristics, natural history and real-world clinical management of ATTR amyloidosis patients * Characterize the safety and effectiveness of patisiran and vutrisiran as part of routine clinical practice in the real-world clinical setting * Describe disease emergence/progression in pre-symptomatic carriers of a known disease-causing transthyretin (TTR) variant

Ages not specified40 locations

View full study

Looking for volunteersRecently updatedNCT07358078

DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting

For Transthyretin Amyloidosis With Cardiomyopathy

The purpose of this study is to: * Describe the clinical characteristics of adult patients with transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM) treated with vutrisiran in routine clinical care * Describe treatment patterns of adult patients with ATTR-CM treated with vutrisiran in routine clinical care * Assess health-related quality of life (HRQOL) in adult patients with ATTR-CM treated with vutrisiran in routine clinical care * Assess healthcare resource use (HCRU) in adult patients with ATTR-CM treated with vutrisiran in routine clinical care * Compare the long-term effectiveness of vutrisiran versus other approved ATTR-CM treatments in routine clinical care

Ages 18 Years+27 locations

View full study

Looking for volunteersRecently updatedNCT05040373

Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program

For Hereditary Transthyretin-mediated (hATTR) Amyloidosis and Polyneuropathy

The purpose of this study is to collect and evaluate pregnancy outcomes, pregnancy complications, and fetal/neonatal/infant outcomes in women exposed to patisiran-LNP.

Ages not specified7 locations

View full study

Looking for volunteersPhase 3Recently updatedNCT07223203

TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy

Tests Nucresiran, Vutrisiran

For Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy and hATTR-PN

The purpose of this study is to: * Determine the efficacy of nucresiran in patients with hATTR-PN by evaluating the effect on neurologic impairment, quality of life, nutritional status, disability, and gait speed * Demonstrate superiority of nucresiran compared to in-study vutrisiran with respect to serum transthyretin (TTR) levels

Ages 18 Years - 85 Years61 locations

View full study

Looking for volunteersRecently updatedNCT04883905

ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)

For Acute Hepatic Porphyria

This global patient registry is being conducted to characterize the natural history and real-world clinical management of patients with AHP, and to further characterize the real-world safety and effectiveness of givosiran and other approved AHP therapies.

Ages 12 Years+30 locations

View full study

Ask about Alnylam Pharmaceuticals's trials

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Trial Alerts, when a new trial opens or a trial changes
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FDA-approved drugs from Alnylam Pharmaceuticals

From the openFDA database. Tap a drug for side effects, dosing and help paying.

Amvuttra vutrisiran

For ATTR Amyloidosis (Transthyretin Amyloidosis). Approved Jun 13, 2022. Taken by subcutaneous route.

Givlaari givosiran sodium

For Acute Hepatic Porphyria. Approved Nov 20, 2019. Taken by subcutaneous route.

Onpattro patisiran

Approved Aug 10, 2018. Taken by intravenous route.

Oxlumo lumasiran

For Primary Hyperoxaluria. Approved Nov 23, 2020. Taken by subcutaneous route.

More about Alnylam Pharmaceuticals

Translating that science into actual approved medicines took Alnylam roughly 16 years of research and several billion dollars in R&D. Alnylam now sells 4 FDA-approved RNAi medicines, partners sell 2 more that came from its research, Leqvio and Qfitlia, and the company trades on the NASDAQ under the ticker ALNY.

Alnylam's first approval came in 2018, when Onpattro (patisiran) was cleared by the FDA for the polyneuropathy form of hereditary transthyretin (ATTR) amyloidosis, a rare disease where misfolded proteins build up in nerves and organs. Amvuttra (vutrisiran) followed and offered the same family of patients a less frequent injection schedule.

In March 2025 the FDA also approved Amvuttra for the heart form of ATTR amyloidosis, both wild-type and hereditary, in adults. Givlaari (givosiran) treats acute hepatic porphyria, a disease that causes severe pain attacks and neurological symptoms, while Oxlumo (lumasiran) treats primary hyperoxaluria type 1, a condition where the liver overproduces a chemical called oxalate that then damages the kidneys.

In hemophilia, Alnylam developed fitusiran, which Sanofi now markets as Qfitlia. The FDA approved Qfitlia on March 28, 2025 for both hemophilia A and B, with or without inhibitors, making it the first RNAi therapeutic for hemophilia. The drug offers a less frequent dosing alternative to existing factor replacement and bispecific antibody options.

Keep reading about Alnylam Pharmaceuticals

Alnylam also discovered inclisiran, which Novartis markets as Leqvio for adults with high cholesterol who cannot reach their LDL targets on statins alone.

Pipeline programs in development extend RNAi into hereditary hemorrhagic telangiectasia (a rare bleeding disorder caused by abnormal blood vessels) and Huntington's disease (a hereditary neurodegenerative disorder), among others. The strategic thesis has not changed since 2002. Find diseases where silencing a single gene can change the course of the illness, and bring patients a precise medicine that addresses the root cause rather than the symptoms.

Where Alnylam Pharmaceuticals trials take place

The trials above run at 648 study sites in 48 countries. Tap a country to see its states, cities and hospitals.

United States

140▼

Germany

47▼

Japan

46▼

France

38▼

Spain

38▼

United Kingdom

34▼

Italy

30▼

China

21▼

Belgium

19▼

Brazil

19▼

Australia

17▼

Portugal

17▼

Rare diseases Alnylam Pharmaceuticals works on

Each disease page lists every open trial, not only Alnylam Pharmaceuticals's, plus approved treatments.

Acute Hepatic Porphyria

Acute hepatic porphyria (AHP) is a group of 4 rare inherited disorders of heme production in the liver: acute intermittent porphyria, hereditary coproporphyria, variegate porphyria and ALAD...

How common: Symptomatic acute intermittent porphyria, the most common type, affects about 5 to 6 people per million in Europe (NORD cites 5.9 per million; a European prospective study found 5.4 per million), roughly 1 in 170,000 to 1 in 185,000 people, and the rate is thought to be similar in the United States

ATTR Amyloidosis (Transthyretin Amyloidosis)

ATTR amyloidosis is a rare progressive disease where abnormal transthyretin protein accumulates as amyloid deposits in the heart, nerves, and other organs.

How common: Approximately 5,000 to 7,000 new cases diagnosed annually in the U.S

Hemophilia A

Hemophilia A is an X-linked bleeding disorder caused by deficiency or dysfunction of clotting factor VIII.

How common: 1 in 4,000 to 5,000 males worldwide; very rare in females

Hemophilia B

Hemophilia B is an X-linked bleeding disorder caused by deficiency or dysfunction of clotting factor IX.

How common: 1 in 25,000 to 33,000 males; very rare in females

Hereditary Hemorrhagic Telangiectasia

Hereditary Hemorrhagic Telangiectasia is a rare genetic disorder characterized by abnormal blood vessel formation (telangiectasias) and arteriovenous malformations causing nosebleeds...

How common: Approximately 1 in 5,000 to 10,000 people

Huntington Disease

Huntington disease is an autosomal dominant neurodegenerative disorder caused by an expanded CAG trinucleotide repeat in the huntingtin gene.

How common: About 3-7 per 100,000 people of European ancestry, according to MedlinePlus Genetics; less common in people of Japanese, Chinese and African descent

Paroxysmal Nocturnal Hemoglobinuria

Paroxysmal Nocturnal Hemoglobinuria is a rare, life-threatening blood disorder where complement-mediated destruction of red blood cells occurs due to acquired mutations in the PIGA gene.

How common: Estimated at 1 to 5 per million people (MedlinePlus Genetics); NORD gives 0.5 to 1.5 per million

Primary Hyperoxaluria

Primary hyperoxaluria is a rare inherited metabolic disorder in which the liver overproduces oxalate, a substance that combines with calcium to form kidney stones and can eventually cause kidney...

How common: 1 to 3 per million people in Europe and North America

Where to get support

Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Alnylam Pharmaceuticals's own site is alnylam.com, and our help paying for treatment page lists drugmaker programs and charity funds.

NIH GARD - Hemophilia A
Hemophilia A and Hemophilia B and 2 more
National Bleeding Disorders Foundation
Hemophilia A and Hemophilia B
American Porphyria Foundation: Acute Intermittent Porphyria
Acute Hepatic Porphyria
American Porphyria Foundation: AHP Drug Safety Database
Acute Hepatic Porphyria
StatPearls - Transthyretin Amyloid Cardiomyopathy
ATTR Amyloidosis (Transthyretin Amyloidosis)
Amyloidosis Research Consortium
ATTR Amyloidosis (Transthyretin Amyloidosis)
National Organization for Rare Disorders (NORD)
Hereditary Hemorrhagic Telangiectasia
HHT Foundation International
Hereditary Hemorrhagic Telangiectasia

Alnylam Pharmaceuticals

Alnylam Pharmaceuticals pipeline

Alnylam Pharmaceuticals clinical trials

9 trials looking for volunteers now

HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB

TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease

ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)

DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting

Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program

TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy

ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)

FDA-approved drugs from Alnylam Pharmaceuticals

More about Alnylam Pharmaceuticals

Where Alnylam Pharmaceuticals trials take place

Rare diseases Alnylam Pharmaceuticals works on

Where to get support

Other companies working on the same diseases

Questions about Alnylam Pharmaceuticals