Overview
Parisiran is a first in class short interfering RNA for the treatment of patients with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis . It is marketed as Onpattro which is formulated as patisiran within a liposome envelope for better delivery to the liver, where transthyretin is produced. The approval for Onpattro was granted to Alnylam Pharmaceuticals, Inc. in August of 2018. Onpattro has been granted Fast Track, Priority Review and Breakthrough Therapy, and Orphan Drug designations.
Background
Parisiran is a first in class short interfering RNA for the treatment of patients with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis . It is marketed as Onpattro which is formulated as patisiran within a liposome envelope for better delivery to the liver, where transthyretin is produced. The approval for Onpattro was granted to Alnylam Pharmaceuticals, Inc. in August of 2018. Onpattro has been granted Fast Track, Priority Review and Breakthrough Therapy, and Orphan Drug designations.
Indication
适用于治疗成人遗传性转甲状腺素介导的淀粉样变性的多发性神经病，如转甲状腺素蛋白淀粉样变性多发性神经病（ATTR-PN）或转甲状腺素蛋白淀粉样变性心肌病（ATTR-CM）。ATTR疾病也属于罕见病。
Associated Conditions
- Hereditary transthyretin-mediated amyloidosis
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
|---|---|---|---|---|---|
2022/08/18 | N/A | NO_LONGER_AVAILABLE | |||
2021/08/27 | Early Phase 1 | Active, not recruiting | Austin Neuromuscular Center | ||
2019/12/17 | N/A | Completed | |||
2019/06/25 | Phase 3 | Active, not recruiting | |||
2019/03/05 | Phase 3 | Completed | |||
2018/11/30 | Phase 3 | Active, not recruiting | |||
2016/10/20 | N/A | APPROVED_FOR_MARKETING | |||
2015/07/29 | Phase 3 | Completed | |||
2014/02/03 | Phase 1 | Completed | |||
2013/10/10 | Phase 3 | Completed |
FDA Drug Approvals
Approved Product | Manufacturer | NDC Code | Route | Strength | Effective Date |
|---|---|---|---|---|---|
| No FDA approvals found for this drug. | |||||
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
|---|---|---|---|
| No EMA approvals found for this drug. | |||
HSA Drug Approvals
Approved Product | Manufacturer | Approval Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No HSA approvals found for this drug. | |||||
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
|---|---|---|---|---|---|
| No NMPA approvals found for this drug. | |||||
PPB Drug Approvals
Approved Product | Registration No. | Company | Licence No. | Strength | Registration Date |
|---|---|---|---|---|---|
| No PPB approvals found for this drug. | |||||
TGA Drug Approvals
Approved Product | ARTG ID | Sponsor | Registration Type | Status | Registration Date |
|---|---|---|---|---|---|
| No TGA approvals found for this drug. | |||||