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Vosoritide

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Also known as: Voxzogo, Bmn 111, Bmn-111, Vosoritide [usan:inn], Unii-7se5582q2p, 1480724-61-5

Molecular Formula

C₁₇₆H₂₉₀N₅₆O₅₁S₃

Molecular Weight

4103 g/mol

InChI Key

IGYWDDBBJPSOTG-WBAGYEQSSA-N

Achondroplasia is an autosomal dominant genetic disease and the most common cause of dwarfism in humans. It results from a gain-of-function missense mutation in FGFR3 that results in a dramatic suppression of bone growth, both in volume and in length. Treatment for achondroplasia includes both surgical and pharmacological interventions, the latter of which includes C-type natriuretic peptide (CNP) analogs. Endogenous CNP, first described in 1998, is primarily responsible for the stimulation of chondrocytes and long bone growth via activity at the NPR-B receptor, making it an attractive target in the treatment of a condition like achondroplasia. While the remarkably short half-life of endogenous CNP - 2 to 3 minutes due to its rapid degradation by endopeptidases - makes it ineffective as a therapeutic intervention, the development of a peptidase-resistant formulation has allowed for its use as a viable treatment option in achondroplasia. Vosoritide is an analog of CNP with proline-glycine on its N-terminus to convey resistance to neutral endopeptidase. It was approved for use under the brand name Voxzogo (BioMarin Pharmaceutical Inc.) in the EU in August 2021 and the US in November 2021, becoming the first pharmacological intervention approved for the treatment of achondroplasia in both regions.

1 2D Structure

Vosoritide

2 Identification

2.1 Computed Descriptors

2.1.1 IUPAC Name

(4R,10S,16S,19S,22S,28S,31S,34S,37S,40S,43S,49S,52R)-52-[[2-[[(2S)-6-amino-2-[[(2S)-2-[[(2S)-2-[[2-[[(2S)-6-amino-2-[[(2S)-6-amino-2-[[(2S)-4-amino-2-[[(2S)-2-[[2-[[(2S)-6-amino-2-[[(2S)-2-[[(2S)-6-amino-2-[[(2S)-2-[[(2S)-2-[[(2S)-4-amino-2-[[(2S)-1-[(2S)-2-[[(2S)-2-[[(2S)-5-amino-5-oxo-2-[[2-[[(2S)-pyrrolidine-2-carbonyl]amino]acetyl]amino]pentanoyl]amino]-4-carboxybutanoyl]amino]-3-(1H-imidazol-5-yl)propanoyl]pyrrolidine-2-carbonyl]amino]-4-oxobutanoyl]amino]propanoyl]amino]-5-carbamimidamidopentanoyl]amino]hexanoyl]amino]-3-(4-hydroxyphenyl)propanoyl]amino]hexanoyl]amino]acetyl]amino]propanoyl]amino]-4-oxobutanoyl]amino]hexanoyl]amino]hexanoyl]amino]acetyl]amino]-4-methylpentanoyl]amino]-3-hydroxypropanoyl]amino]hexanoyl]amino]acetyl]amino]-40-(4-aminobutyl)-49-benzyl-28-[(2S)-butan-2-yl]-31-(3-carbamimidamidopropyl)-34-(carboxymethyl)-16,22-bis(hydroxymethyl)-10,37,43-tris(2-methylpropyl)-19-(2-methylsulfanylethyl)-6,9,12,15,18,21,24,27,30,33,36,39,42,45,48,51-hexadecaoxo-1,2-dithia-5,8,11,14,17,20,23,26,29,32,35,38,41,44,47,50-hexadecazacyclotripentacontane-4-carboxylic acid

2.1.2 InChI

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

2.1.3 InChI Key

IGYWDDBBJPSOTG-WBAGYEQSSA-N

2.1.4 Canonical SMILES

CCC(C)C1C(=O)NCC(=O)NC(C(=O)NC(C(=O)NC(C(=O)NCC(=O)NC(C(=O)NCC(=O)NC(CSSCC(C(=O)NC(C(=O)NCC(=O)NC(C(=O)NC(C(=O)NC(C(=O)NC(C(=O)NC(C(=O)N1)CCCNC(=N)N)CC(=O)O)CC(C)C)CCCCN)CC(C)C)CC2=CC=CC=C2)NC(=O)CNC(=O)C(CCCCN)NC(=O)C(CO)NC(=O)C(CC(C)C)NC(=O)CNC(=O)C(CCCCN)NC(=O)C(CCCCN)NC(=O)C(CC(=O)N)NC(=O)C(C)NC(=O)CNC(=O)C(CCCCN)NC(=O)C(CC3=CC=C(C=C3)O)NC(=O)C(CCCCN)NC(=O)C(CCCNC(=N)N)NC(=O)C(C)NC(=O)C(CC(=O)N)NC(=O)C4CCCN4C(=O)C(CC5=CN=CN5)NC(=O)C(CCC(=O)O)NC(=O)C(CCC(=O)N)NC(=O)CNC(=O)C6CCCN6)C(=O)O)CC(C)C)CO)CCSC)CO

2.1.5 Isomeric SMILES

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)CC2=CC=CC=C2)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](CC3=CC=C(C=C3)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](CC5=CN=CN5)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

2.2 Synonyms

2.2.1 MeSH Synonyms

1. Bmn 111

2. Voxzogo

2.2.2 Depositor-Supplied Synonyms

1. Voxzogo

2. Bmn 111

3. Bmn-111

4. Vosoritide [usan:inn]

5. Unii-7se5582q2p

6. 1480724-61-5

7. Gtpl9068

8. 7se5582q2p

2.3 Create Date

2016-05-29

3 Chemical and Physical Properties

Molecular Formula	C₁₇₆H₂₉₀N₅₆O₅₁S₃
Molecular Weight	4103 g/mol
XLogP3	-20.6
Hydrogen Bond Donor Count	61
Hydrogen Bond Acceptor Count	64
Rotatable Bond Count	112
Exact Mass	4101.1016174 g/mol
Monoisotopic Mass	4100.0982626 g/mol
Topological Polar Surface Area	1820 Å²
Heavy Atom Count	286
Formal Charge	0
Complexity	9600
Isotope Atom Count	0
Defined Atom Stereocenter Count	32
Undefined Atom Stereocenter Count	0
Defined Bond Stereocenter Count	0
Undefined Bond Stereocenter Count	0
Covalently Bonded Unit Count	1

4 Drug and Medication Information

4.1 Drug Indication

Vosoritide is indicated for the promotion of linear growth in pediatric patients with achondroplasia who are 5 years of age and older with open epiphyses.

Voxzogo is indicated for the treatment of achondroplasia in patients 2 years of age and older whose epiphyses are not closed. The diagnosis of achondroplasia should be confirmed by appropriate genetic testing.

5 Pharmacology and Biochemistry

5.1 Pharmacology

Vosoritide is an analog of C-type natriuretic peptide that promotes bone growth to combat growth suppression in children with achondroplasia. Urinary cyclic guanosine monophosphate (cGMP) and serum collagen type X marker (CXM) are both elevated following daily therapy with vosoritide and serve as biomarkers for evidence of increased endochondral bone growth, with cGMP indicative of NPR-B binding activity and CXM indicative of bone metabolism. Although relatively well-tolerated, transient episodes of hypotension have been observed in clinical studies. Patients with pre-existing cardiovascular disease and those taking antihypertensive medications were excluded from clinical trials. The risk of hypotension may be reduced by ensuring adequate food and fluid intake prior to the administration of vosoritide. The use of vosoritide in patients with an eGFR <60 mL/min/1.73m² should also be avoided as there are no data on the influence of renal impairment on its pharmacokinetics.

5.2 ATC Code

M05BX

M - Musculo-skeletal system

M05 - Drugs for treatment of bone diseases

M05B - Drugs affecting bone structure and mineralization

M05BX - Other drugs affecting bone structure and mineralization

M05BX07 - Vosoritide

5.3 Absorption, Distribution and Excretion

Absorption

In patients receiving daily subcutaneous injections of vosoritide 15 mcg/kg, the mean C_max ranged from 4.71-7.18 ng/mL and the mean AUC_{0-_t} ranged from 161-290 ng-min/mL. The median T_max following subcutaneous injection was approximately 15 minutes.

Volume of Distribution

The mean apparent volume of distribution following the subcutaneous administration of 15 mcg/kg of vosoritide ranged from 2880 to 3020 mL/kg.

Clearance

The mean apparent clearance following the subcutaneous administration of 15 mcg/kg of vosoritide ranged from 79.4 to 104 mL/min/kg.

5.4 Metabolism/Metabolites

As with other therapeutic proteins, vosoritide is likely metabolized via catabolic pathways into smaller peptides and amino acids.

5.5 Biological Half-Life

The mean half-life following the subcutaneous administration of 15 mcg/kg of vosoritide ranged from 21.0 to 27.9 minutes.

5.6 Mechanism of Action

Achondroplasia is a congenital disease resulting from a missense mutation in the fibroblast growth factor receptor 3 (_FGFR3_) gene, resulting in a gain-of-function that negatively regulates endochondral bone growth. Under normal conditions, _FGFR3_ is expressed during both embryonic and postnatal development, but serves a different role in each. During initial development, FGFR3 signaling promotes proliferation of chondrocytes (i.e. growth), whereas postnatal skeletal growth is actually inhibited by FGFR3 - as a result, the pathologic activation of FGFR3 observed in patients with achondroplasia leads to suppressed pre-pubertal skeletal growth. Vosoritide is an analog of C-type natriuretic peptide (CNP), a signaling molecule that appears primarily responsible for the stimulation of chondrocytes and the growth of long bones. The binding of CNP (or vosoritide) with its corresponding receptor, NPR-B, results in a signaling cascade that ultimately inhibits the MAPK/ERK pathway via inhibition of RAF-1 and stimulates the proliferation and differentiation of chondrocytes. This activity serves to antagonize the downstream signaling resulting from FGFR3 and its resultant effects on bone growth.

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Aspen API is the cooperative entity formed by Aspen Oss in the Netherlands and Fine Chemicals Corporation in South Africa. With quality, compliance, and teamwork at its core, Aspen API works tirelessly to deliver top-quality APIs to clients worldwide. Aspen API has a portfolio of over 58 high-quality APIs, including high potency, oncology, peptides, narcotics, analgesics, botanical extractions, and biochemicals. Aspen API places a strong emphasis on sustainability. It employs eco-friendly technologies and solvents in its chemical processes, ensuring responsible and environmentally conscious manufacturing.