Givosiran

Givosiran
Clinical data
Trade names	Givlaari
License data	US DailyMed: Givosiran;
Routes of; administration	Subcutaneous injection
ATC code	None;
Legal status
Legal status	US: ℞-only ;
Identifiers
	IUPAC name N-[1,3-bis[3-[3-[5-[(2R,3R,4R,5R,6R)-3-acetamido-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxypentanoylamino]propylamino]-3-oxopropoxy]-2-[[3-[3-[5-[(2R,3R,4R,5R,6R)-3-acetamido-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxypentanoylamino]propylamino]-3-oxopropoxy]methyl]propan-2-yl]-12-[(2R,4R)-4-hydroxy-2-methylpyrrolidin-1-yl]-12-oxododecanamide;
CAS Number	1639325-43-1;
PubChem CID	119058042;
UNII	ROV204583W;
KEGG	D11702;
Chemical and physical data
Formula	C524H694F16N173O316P43S6
Molar mass	16300.42 g·mol−1
3D model (JSmol)	Interactive image;
	SMILES C[C@@H]1C[C@H](CN1C(=O)CCCCCCCCCCC(=O)NC(COCCC(=O)NCCCNC(=O)CCCCO[C@H]2[C@@H]([C@H]([C@H]([C@H](O2)CO)O)O)NC(=O)C)(COCCC(=O)NCCCNC(=O)CCCCO[C@H]3[C@@H]([C@H]([C@H]([C@H](O3)CO)O)O)NC(=O)C)COCCC(=O)NCCCNC(=O)CCCCO[C@H]4[C@@H]([C@H]([C@H]([C@H](O4)CO)O)O)NC(=O)C)O;
	InChI InChI=1S/C78H139N11O30/c1-50-42-54(96)43-89(50)65(104)26-12-10-8-6-5-7-9-11-25-64(103)88-78(47-111-39-27-61(100)82-33-19-30-79-58(97)22-13-16-36-114-75-66(85-51(2)93)72(108)69(105)55(44-90)117-75,48-112-40-28-62(101)83-34-20-31-80-59(98)23-14-17-37-115-76-67(86-52(3)94)73(109)70(106)56(45-91)118-76)49-113-41-29-63(102)84-35-21-32-81-60(99)24-15-18-38-116-77-68(87-53(4)95)74(110)71(107)57(46-92)119-77/h50,54-57,66-77,90-92,96,105-110H,5-49H2,1-4H3,(H,79,97)(H,80,98)(H,81,99)(H,82,100)(H,83,101)(H,84,102)(H,85,93)(H,86,94)(H,87,95)(H,88,103)/t50-,54-,55-,56-,57-,66-,67-,68-,69+,70+,71+,72-,73-,74-,75-,76-,77-/m1/s1; Key:RUPXJRIDSUCQAN-PQNNUJSWSA-N;

Givosiran, sold under the brand name Givlaari, is for the treatment of adults with acute hepatic porphyria, 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).[1][2] Givosiran is a small interfering RNA (siRNA) directed towards 5-aminolevulinic acid synthase, an important enzyme in the production of heme.[3]

History

In November 2019, givosiran was approved in the United States for the treatment of adults with acute hepatic porphyria (AHP).[1][4]

The U.S. Food and Drug Administration (FDA) granted the application for givosiran breakthrough therapy designation, priority review designation, and orphan drug designation.[1] The FDA granted the approval of Givlaari to Alnylam Pharmaceuticals.[1]

gollark: Ah, no, you want wireless specifically, but peripheral.find can still be used that way.

gollark: At least the `peripheral.find("modem", rednet.open)` bit.

gollark: Possibly.

gollark: Also, `local modem = peripheral.find("modem", rednet.open) ~= nil` could replace all your modem finding logic.

gollark: There's a perfectly good keys library.

References

"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.
https://www.drugbank.ca/drugs/DB15066
"Drug Trials Snapshots: Givlaari". U.S. Food and Drug Administration (FDA). 20 November 2019. Retrieved 24 January 2020. 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).
"Givosiran". Drug Information Portal. DrugBank.

This article is issued from Wikipedia. The text is licensed under Creative Commons - Attribution - Sharealike. Additional terms may apply for the media files.

[FDA_PR-1] "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_PR_2-2] "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.

[3] ttps://www.drugbank.ca/drugs/DB15066

[FDA_Snapshot-4] "Drug Trials Snapshots: Givlaari". U.S. Food and Drug Administration (FDA). 20 November 2019. Retrieved 24 January 2020. This article incorporates text from this source, which is in the public domain.