Givosiran
As of the edition of 20 October 2026, Givosiran is an oligonucleotide: marketed, first approved in 2019 (US); acts on ALAS1; no US protection is listed.
Sources: FDA Orange Book and Purple Book, other approval registers, ClinicalTrials.gov, ChEMBL.
The drug
- Status
- Marketed; latest approval 2021
- Kind
- Oligonucleotide
- Brand name
- Givlaari
- First approved
- 20 November 2019 (US)
- Approvals on record
- 5 in 4 regions
- Holder of the most recent approval
- Alnylam Pharmaceuticals; 3 holders in all
- Registered trials
- 4, 0 stopped early
- Disease areas
- Digestive system; Inherited and neonatal; Nutrition and metabolism; Skin and connective tissue
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.
Companies that need a new molecule because of this drug
1 company on route B because of this drug.
Companies on route B for this drug
In the product1 company is on route B because of Givosiran.
See the 1 company on route B for this drug, with the facts behind eachRequest access. Nothing is shown until you do.
Target
| Gene | Protein | UniProt |
|---|---|---|
| ALAS1 | 5-aminolevulinate synthase, non-specific, mitochondrial | P13196 |
Registered trials
| Phase | Trials | Completed | Active | Stopped early |
|---|---|---|---|---|
| Phase not stated | 1 | 0 | 0 | 0 |
| Phase 1 | 1 | 1 | 0 | 0 |
| Phase 2 | 1 | 1 | 0 | 0 |
| Phase 3 | 1 | 1 | 0 | 0 |
| All trials | 4 |
Who has held its approvals
| Company | Role | Years |
|---|---|---|
| Alnylam Pharmaceuticals | Originator, Applicant and Lead sponsor of trials | 2019 to now |