Basic Information
Onpattro
Regulatory Information
EMEA/H/C/004699
August 27, 2018
July 26, 2018
14
March 25, 2025
Company Information
Netherlands
Antonio Vivaldistraat 150 1083 HP Amsterdam
Alnylam Netherlands BV
Drug Classification
Active Substances Detail
EMA Resources
Detailed Information
Therapeutic Indication
### Therapeutic indication Onpattro is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy.
Overview Summary
Onpattro is a medicine used to treat nerve damage caused by hereditary transthyretin (hATTR) amyloidosis, a disease in which abnormal proteins called amyloids build up in tissues around the body including around the nerves. Onpattro is used in adult patients in the first two stages of the nerve damage (stage 1, when the patient is able to walk unaided, and stage 2, when the patient can still walk but needs help). hATTR amyloidosis is rare, and Onpattro was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 15 April 2011. Onpattro contains the active substance patisiran.