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Givosiran

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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]

Givosiran
Clinical data
Trade namesGivlaari
License data
Routes of
administration		Subcutaneous injection
ATC code
  • None
Legal status
Legal status
Identifiers
  • 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
PubChem CID
UNII
KEGG
Chemical and physical data
FormulaC524H694F16N173O316P43S6
Molar mass16300.42 g·mol−1
3D model (JSmol)
  • 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=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

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]

References

  1. ^ a b c d "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. Public Domain  This article incorporates text from this source, which is in the public domain.
  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.Public Domain  This article incorporates text from this source, which is in the public domain.
  3. ^ https://www.drugbank.ca/drugs/DB15066
  4. ^ "Drug Trials Snapshots: Givlaari". U.S. Food and Drug Administration (FDA). 20 November 2019. Retrieved 24 January 2020. Public Domain  This article incorporates text from this source, which is in the public domain.


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