Alnylam to Present Detailed Results from the HELIOS-B Phase 3 Study of Vutrisiran▼ in Patients with ATTR Amyloidosis with Cardiomyopathy at the European Society of Cardiology Congress 2024 - Seite 2

About AMVUTTRA (vutrisiran)

AMVUTTRA (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of mutant and wild‑type transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. Administered quarterly via subcutaneous injection, vutrisiran is approved and marketed in more than 15 countries for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. In the UK, vutrisiran is specifically indicated for the treatment of hATTR in adult patients with stage 1 or stage 2 polyneuropathy. Vutrisiran is also in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM), which encompasses both wild-type and hereditary forms of the disease. For US Media only: For more information about vutrisiran, including the full U.S. Prescribing Information, visit AMVUTTRA.com. For UK Media: For the UK Summary of Product Characteristics, see https://www.medicines.org.uk/emc/product/14060.

About ONPATTRO (patisiran)

ONPATTRO (patisiran) is an RNAi therapeutic designed to silence TTR messenger RNA and reduce the production of TTR protein in the liver. Administered intravenously, patisiran is approved in forty countries for the treatment of the polyneuropathy of hereditary transthyretin-amyloidosis (hATTR-PN) in adults. For US Media only: For more information about patisiran, including the full U.S. Prescribing Information, visit ONPATTRO.com. For UK Media: For the UK Summary of Product Characteristics, see https://www.medicines.org.uk/emc/product/10368.

About ATTR

Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy, or both manifestations of disease. There are two different forms of ATTR – hereditary ATTR (hATTR), which is caused by a TTR gene variant and affects approximately 50,000 people worldwide, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant and impacts an estimated 200,000 – 300,000 people worldwide.