Chemistry:Givosiran
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| Trade names | Givlaari |
| AHFS/Drugs.com | Monograph |
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| Routes of administration | Subcutaneous injection |
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| Formula | C524H694F16N173O316P43S6 |
| Molar mass | 16300.42 g·mol−1 |
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Givosiran, sold under the brand name Givlaari, is for the treatment of adults with acute hepatic porphyria,[1] a genetic disorder resulting in the buildup of toxic porphyrin molecules which are formed during the production of heme (which helps bind oxygen in the blood).[2][3] Givosiran is a small interfering RNA (siRNA) directed towards delta-aminolevulinate synthase 1 (ALAS1)[4], an important enzyme in the production of heme.
The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[5]
History
In November 2019, givosiran was approved in the United States for the treatment of adults with acute hepatic porphyria (AHP).[2][6]
The U.S. Food and Drug Administration (FDA) granted the application for givosiran breakthrough therapy designation, priority review designation, and orphan drug designation.[2] The FDA granted the approval of Givlaari to Alnylam Pharmaceuticals.[2]
References
- ↑ "Givosiran: A Review in Acute Hepatic Porphyria". Drugs 81 (7): 841–848. May 2021. doi:10.1007/s40265-021-01511-3. PMID 33871817.
- ↑ 2.0 2.1 2.2 2.3 "FDA approves first treatment for inherited rare disease". U.S. Food and Drug Administration (FDA) (Press release). 20 November 2019. Archived from the original on 21 November 2019. Retrieved 20 November 2019.
This article incorporates text from this source, which is in the public domain.
- ↑ "FDA approves givosiran for acute hepatic porphyria". U.S. Food and Drug Administration (FDA) (Press release). 20 November 2019. Archived from the original on 21 November 2019. Retrieved 20 November 2019. This article incorporates text from this source, which is in the public domain.
- ↑ Balwani M, Sardh E, et al.; ENVISION Investigators. Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria. N Engl J Med. 2020 Jun 11;382(24):2289-2301. doi: 10.1056/NEJMoa1913147. PMID: 32521132.
- ↑ "New Drug Therapy Approvals 2019". 31 December 2019. https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/new-drug-therapy-approvals-2019.
- ↑ "Drug Trials Snapshots: Givlaari". 20 November 2019. http://www.fda.gov/drugs/drug-trials-snapshots-givlaari. This article incorporates text from this source, which is in the public domain.
External links
- "Givosiran". Drug Information Portal. U.S. National Library of Medicine (NLM). https://druginfo.nlm.nih.gov/drugportal/name/givosiran.
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