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User:Mr. Ibrahem/Givosiran

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Mr. Ibrahem/Givosiran
Clinical data
Trade namesGivlaari
AHFS/Drugs.comMonograph
License data
Routes of
administration		Subcutaneous injection
Drug classSmall interfering RNA (siRNA)[1]
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
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

Givosiran, sold under the brand name Givlaari, is a medication used to treat acute hepatic porphyria (AHP).[1] It is given by injection under the skin.[1] It is used in people over the age of 11.[1]

Common side effects include pain at the site of injection, nausea, and tiredness.[1] Other side effects may include anaphylaxis, kidney problems, and liver problems.[2] It is a small interfering RNA (siRNA) that decreases the amount of the enzyme aminolevulinic acid synthase 1 (ALAS1) and therefore decreases the production of heme.[1][3]

Givosiran was approved for medical use in the United States in 2019 and Europe in 2020.[2][1] In the United Kingdom a 189 mg vial costs the NHS about £4200 as of 2021.[3] This amount in the United States costs about 40,700 USD.[4]

References[edit]

  1. ^ a b c d e f g h i "Givlaari". Archived from the original on 12 November 2021. Retrieved 3 December 2021.
  2. ^ a b c "Givosiran Monograph for Professionals". Drugs.com. Archived from the original on 26 October 2021. Retrieved 3 December 2021.
  3. ^ a b "Givosiran". SPS - Specialist Pharmacy Service. 18 June 2018. Archived from the original on 31 July 2020. Retrieved 3 December 2021.
  4. ^ "Givlaari Prices, Coupons & Patient Assistance Programs". Drugs.com. Archived from the original on 22 January 2021. Retrieved 3 December 2021.
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