Alnylam Pharmaceuticals
Alnylam Pharmaceuticals works on 18 rare diseases tracked on Trial Friend, including Alpha-1 Antitrypsin Deficiency, ATTR Amyloidosis (Transthyretin Amyloidosis), Atypical Hemolytic Uremic Syndrome and 15 more, with 13 recruiting clinical trials and 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. Translating that science into actual approved medicines took Alnylam roughly 16 years of research and several billion dollars in R&D. The company now has six approved drugs across a portfolio of rare and chronic diseases and 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. 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. 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.
Alnylam Pharmaceuticals Drug Pipeline
Alnylam Pharmaceuticals has 13 active clinical trials across 4 development stages, with 13 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline includes all of Alnylam Pharmaceuticals's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.
Alnylam Pharmaceuticals Clinical Trials (13)
Active and recruiting clinical trials sponsored by Alnylam Pharmaceuticals, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
Alnylam Pharmaceuticals FDA-Approved Drugs (4)
Medications developed or marketed by Alnylam Pharmaceuticals that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.
| Drug Name | Brand Name | Rare Diseases | Approval Date |
|---|---|---|---|
| VUTRISIRAN | AMVUTTRA subcutaneous | Jun 13, 2022 | |
| GIVOSIRAN SODIUM | GIVLAARI subcutaneous | Nov 20, 2019 | |
| PATISIRAN | Onpattro intravenous | — | Aug 10, 2018 |
| LUMASIRAN | OXLUMO subcutaneous | Nov 23, 2020 |
Alnylam Pharmaceuticals Trial Locations
Alnylam Pharmaceuticals clinical trials are running at 619 sites in 47 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (18)
Diseases targeted by Alnylam Pharmaceuticals's clinical trial and drug development programs
Alpha-1 antitrypsin deficiency is a genetic disorder affecting the lungs and liver, caused by insufficient production of the protective enzyme alpha-1 antitrypsin. Without adequate protection, neutrop...
ATTR amyloidosis is a rare progressive disease where abnormal transthyretin protein accumulates as amyloid deposits in the heart, nerves, and other organs. It can be inherited (hereditary ATTR) or dev...
Atypical Hemolytic Uremic Syndrome (aHUS) is a rare kidney disease caused by uncontrolled activation of the complement system, a part of the immune system that normally helps fight infection. This act...
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) is an autoimmune disorder affecting the peripheral nerves, causing progressive weakness and impaired function in the legs and arms. The immune ...
Guillain-Barré syndrome is a rare autoimmune neurological disorder in which the body's immune system attacks the peripheral nerves, causing rapidly progressive muscle weakness that typically starts in...
Hemophilia A is an X-linked bleeding disorder caused by deficiency or dysfunction of clotting factor VIII. Severity depends on factor levels, ranging from mild to severe hemorrhage. Modern factor repl...
Patient Resources
Organizations and resources related to Alnylam Pharmaceuticals's rare disease focus areas
Frequently Asked Questions About Alnylam Pharmaceuticals
Common questions about Alnylam Pharmaceuticals's rare disease programs, clinical trials, and treatments.