Patisiran
As of the edition of 20 October 2026, Patisiran is an oligonucleotide: marketed, first approved in 2018 (US); acts on TTR; no US protection is listed.
Sources: FDA Orange Book and Purple Book, other approval registers, ClinicalTrials.gov, ChEMBL.
The drug
- Status
- Marketed; latest approval 2020
- Kind
- Oligonucleotide
- Brand name
- Onpattro
- First approved
- 10 August 2018 (US)
- Approvals on record
- 5 in 4 regions
- Holder of the most recent approval
- SPECIALTY PHARMA GOIAS LTDA; 3 holders in all
- Registered trials
- 9, 0 stopped early
- Disease areas
- Nutrition and metabolism; Inherited and neonatal; Nervous system; Heart and blood vessels
US protection
No US protection is listed.
The FDA registers list no drug-substance patent or exclusivity that counts for this drug.
This is the US only.
Target
| Gene | Protein | UniProt |
|---|---|---|
| TTR | Transthyretin | P02766 |
Registered trials
| Phase | Trials | Completed | Active | Stopped early |
|---|---|---|---|---|
| Phase not stated | 3 | 1 | 0 | 0 |
| Phase 1 | 1 | 1 | 0 | 0 |
| Phase 2 | 2 | 2 | 0 | 0 |
| Phase 3 | 3 | 3 | 0 | 0 |
| All trials | 9 |
Who has held its approvals
| Company | Role | Years |
|---|---|---|
| Alnylam Pharmaceuticals | Originator, Applicant and Lead sponsor of trials | 2018 to now |