Vosoritide

Vosoritide
Clinical data
Trade namesVoxzogo
Other namesBMN-111
License data
Pregnancy
category
Routes of
administration
Subcutaneous injection
ATC code
Legal status
Legal status
Identifiers
CAS Number
DrugBank
ChemSpider
UNII
KEGG
Chemical and physical data
FormulaC176H290N56O51S3
Molar mass4102.78 g·mol−1
3D model (JSmol)
  • CC[C@H](C)[C@H]1C(=O)NCC(=O)N[C@H](C(=O)N[C@H](C(=O)N[C@H](C(=O)NCC(=O)N[C@H](C(=O)NCC(=O)N[C@@H](CSSC[C@@H](C(=O)N[C@H](C(=O)NCC(=O)N[C@H](C(=O)N[C@H](C(=O)N[C@H](C(=O)N[C@H](C(=O)N[C@H](C(=O)N1)CCCNC(=N)N)CC(=O)O)CC(C)C)CCCCN)CC(C)C)Cc2ccccc2)NC(=O)CNC(=O)[C@H](CCCCN)NC(=O)[C@H](CO)NC(=O)[C@H](CC(C)C)NC(=O)CNC(=O)[C@H](CCCCN)NC(=O)[C@H](CCCCN)NC(=O)[C@H](CC(=O)N)NC(=O)[C@H](C)NC(=O)CNC(=O)[C@H](CCCCN)NC(=O)[C@H](Cc3ccc(cc3)O)NC(=O)[C@H](CCCCN)NC(=O)[C@H](CCCNC(=N)N)NC(=O)[C@H](C)NC(=O)[C@H](CC(=O)N)NC(=O)[C@@H]4CCCN4C(=O)[C@H](Cc5cnc[nH]5)NC(=O)[C@H](CCC(=O)O)NC(=O)[C@H](CCC(=O)N)NC(=O)CNC(=O)[C@@H]6CCCN6)C(=O)O)CC(C)C)CO)CCSC)CO
  • InChI=1S/C176H290N56O51S3/c1-14-95(10)143-172(280)201-84-138(245)209-125(86-234)168(276)221-113(54-65-284-13)159(267)229-124(85-233)152(260)200-81-135(242)206-114(66-91(2)3)150(258)198-83-140(247)211-128(174(282)283)89-286-285-88-127(170(278)224-118(70-98-34-16-15-17-35-98)151(259)199-80-137(244)207-115(67-92(4)5)162(270)217-108(41-23-29-60-182)155(263)223-117(69-94(8)9)164(272)226-122(75-142(250)251)167(275)219-110(160(268)231-143)44-32-63-193-176(188)189)210-139(246)82-197-149(257)105(38-20-26-57-179)215-169(277)126(87-235)230-163(271)116(68-93(6)7)208-136(243)79-196-147(255)103(36-18-24-55-177)213-153(261)106(39-21-27-58-180)218-166(274)121(74-132(185)239)222-144(252)96(11)203-133(240)77-195-148(256)104(37-19-25-56-178)214-165(273)119(71-99-46-48-101(236)49-47-99)225-156(264)107(40-22-28-59-181)216-154(262)109(43-31-62-192-175(186)187)212-145(253)97(12)204-161(269)120(73-131(184)238)227-171(279)129-45-33-64-232(129)173(281)123(72-100-76-190-90-202-100)228-158(266)112(51-53-141(248)249)220-157(265)111(50-52-130(183)237)205-134(241)78-194-146(254)102-42-30-61-191-102/h15-17,34-35,46-49,76,90-97,102-129,143,191,233-236H,14,18-33,36-45,50-75,77-89,177-182H2,1-13H3,(H2,183,237)(H2,184,238)(H2,185,239)(H,190,202)(H,194,254)(H,195,256)(H,196,255)(H,197,257)(H,198,258)(H,199,259)(H,200,260)(H,201,280)(H,203,240)(H,204,269)(H,205,241)(H,206,242)(H,207,244)(H,208,243)(H,209,245)(H,210,246)(H,211,247)(H,212,253)(H,213,261)(H,214,273)(H,215,277)(H,216,262)(H,217,270)(H,218,274)(H,219,275)(H,220,265)(H,221,276)(H,222,252)(H,223,263)(H,224,278)(H,225,264)(H,226,272)(H,227,279)(H,228,266)(H,229,267)(H,230,271)(H,231,268)(H,248,249)(H,250,251)(H,282,283)(H4,186,187,192)(H4,188,189,193)/t95-,96-,97-,102-,103-,104-,105-,106-,107-,108-,109-,110-,111-,112-,113-,114-,115-,116-,117-,118-,119-,120-,121-,122-,123-,124-,125-,126-,127-,128-,129-,143-/m0/s1
  • Key:IGYWDDBBJPSOTG-WBAGYEQSSA-N

Vosoritide, sold under the brand name Voxzogo, is a medication used for the treatment of achondroplasia,[3][4][5] a genetic condition that causes severely short stature and disproportionate growth.[4]

Achondroplasia is caused by a genetic mutation that increases the activity of a certain growth regulation gene called fibroblast growth factor receptor 3 (FGFR3). The overabundance of protein coded by the FGFR3 gene prevents normal bone growth.[4] Vosoritide works by binding to a specific receptor called natriuretic peptide receptor B that reduces the growth regulation gene's activity, thus allowing for bone growth.[4]

The most common side effects include injection site reactions (such as swelling, redness, itching, or pain), vomiting, and decreased blood pressure.[3][4][5]

Vosoritide was approved for medical use in the European Union in August 2021,[5][6][7] and in the United States in November 2021.[4][8] The US Food and Drug Administration considers it to be a first-in-class medication.[9]

  1. ^ a b "Voxzogo APMDS". Therapeutic Goods Administration (TGA). 4 August 2022. Archived from the original on 6 August 2022. Retrieved 6 August 2022.
  2. ^ "Updates to the Prescribing Medicines in Pregnancy database". Therapeutic Goods Administration (TGA). 21 December 2022. Archived from the original on 3 April 2022. Retrieved 2 January 2023.
  3. ^ a b c "Voxzogo 0.4MG- vosoritide kit Voxzogo 0.56MG- vosoritide kit Voxzogo 1.2MG- vosoritide kit". DailyMed. Archived from the original on 19 December 2021. Retrieved 19 December 2021.
  4. ^ a b c d e f g "FDA Approves First Drug to Improve Growth in Children with Most Common Form of Dwarfism". U.S. Food and Drug Administration (FDA) (Press release). 19 November 2021. Archived from the original on 19 November 2021. Retrieved 19 November 2021. Public Domain This article incorporates text from this source, which is in the public domain.
  5. ^ a b c d Cite error: The named reference Voxzogo EPAR was invoked but never defined (see the help page).
  6. ^ "Voxzogo Product information". Union Register of medicinal products. Archived from the original on 4 March 2023. Retrieved 3 March 2023.
  7. ^ "European Commission Approves BioMarin's Voxzogo (vosoritide) for the Treatment of Children with Achondroplasia from Age 2 Until Growth Plates Close" (Press release). BioMarin Pharmaceutical. 27 August 2021. Archived from the original on 28 August 2021. Retrieved 9 September 2021 – via PR Newswire.
  8. ^ "BioMarin Receives FDA Approval for Voxzogo (vosoritide) for Injection, Indicated to Increase Linear Growth in Children with Achondroplasia Aged 5 and Up with Open Growth Plates" (Press release). BioMarin Pharmaceutical. 19 November 2021. Archived from the original on 6 August 2022. Retrieved 19 November 2021 – via PR Newswire.
  9. ^ Advancing Health Through Innovation: New Drug Therapy Approvals 2021 (PDF). U.S. Food and Drug Administration (FDA) (Report). 13 May 2022. Archived from the original on 6 December 2022. Retrieved 22 January 2023. Public Domain This article incorporates text from this source, which is in the public domain.

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