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.
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.
Therapeutic Area (MeSH)
ATC Code
N07
ATC Item
其它神经系统用药
Pharmacotherapeutic Group
Other nervous system drugs
Active Substance (Summary)
INN / Common Names
| Substance | CAS | Monograph |
|---|---|---|
| patisiran sodium | N/A | Patisiran sodium |
EMA Name
Onpattro
Medicine Name
Onpattro
Aliases
N/ANo risk management plan link.
