Vamorolone

Vamorolone
Clinical data
Trade names	Agamree
Other names	VBP; VBP-15; 17α,21-Dihydroxy-16α-methylpregna-1,4,9(11)-triene-3,20-dione
AHFS/Drugs.com	Monograph
MedlinePlus	a624005
License data	US DailyMed: Vamorolone;
Routes of; administration	By mouth
ATC code	H02AB18 (WHO) ;
Legal status
Legal status	US: ℞-only; EU: Rx-only;
Identifiers
	IUPAC name (8S,10S,13S,14S,16R,17R)-17-Hydroxy-17-(2-hydroxyacetyl)-10,13,16-trimethyl-7,8,12,14,15,16-hexahydro-6H-cyclopenta[a]phenanthren-3-one;
CAS Number	13209-41-1;
PubChem CID	3035000;
DrugBank	DB15114;
ChemSpider	2299335;
UNII	8XP29XMB43;
KEGG	D11000;
ChEBI	CHEBI:228304;
ChEMBL	ChEMBL2348780;
CompTox Dashboard (EPA)	DTXSID60927527 ;
ECHA InfoCard	100.032.874
Chemical and physical data
Formula	C22H28O4
Molar mass	356.462 g·mol−1
3D model (JSmol)	Interactive image;
	SMILES C[C@@H]1C[C@H]2[C@@H]3CCC4=CC(=O)C=C[C@@]4(C3=CC[C@@]2([C@]1(C(=O)CO)O)C)C;
	InChI InChI=1S/C22H28O4/c1-13-10-18-16-5-4-14-11-15(24)6-8-20(14,2)17(16)7-9-21(18,3)22(13,26)19(25)12-23/h6-8,11,13,16,18,23,26H,4-5,9-10,12H2,1-3H3/t13-,16-,18+,20+,21+,22+/m1/s1; Key:ZYTXTXAMMDTYDQ-DGEXFFLYSA-N;

Vamorolone, sold under the brand name Agamree, is a synthetic corticosteroid, which is used for the treatment of Duchenne muscular dystrophy.^[4]^[5]^[6]^[7]^[8] It is taken by mouth.^[1] It is a dual atypical glucocorticoid and antimineralocorticoid.^[9]

The most common adverse reactions include cushingoid features, psychiatric disorders, vomiting, increased weight, and vitamin D deficiency.^[10]

Vamorolone was approved for medical use in the United States in October 2023,^[11]^[10] and in the European Union in December 2023.^[2]^[3]

^ ^a ^b "Agamree- vamorolone kit". DailyMed. 26 October 2023. Retrieved 20 November 2023.
^ ^a ^b Cite error: The named reference Agamree EPAR was invoked but never defined (see the help page).
^ ^a ^b "Agamree Product information". Union Register of medicinal products. 15 December 2023. Retrieved 26 December 2023.
^ "Vamorolone - ReveraGen Biopharma". AdisInsight. Springer Nature Switzerland AG. Archived from the original on 7 October 2017. Retrieved 2 July 2017.
^ Reeves EK, Hoffman EP, Nagaraju K, Damsker JM, McCall JM (April 2013). "VBP15: preclinical characterization of a novel anti-inflammatory delta 9,11 steroid". Bioorganic & Medicinal Chemistry. 21 (8): 2241–2249. doi:10.1016/j.bmc.2013.02.009. PMC 4088988. PMID 23498916.
^ Heier CR, Damsker JM, Yu Q, Dillingham BC, Huynh T, Van der Meulen JH, et al. (October 2013). "VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects". EMBO Molecular Medicine. 5 (10): 1569–1585. doi:10.1002/emmm.201302621. PMC 3799580. PMID 24014378.
^ Dadgar S, Wang Z, Johnston H, Kesari A, Nagaraju K, Chen YW, et al. (October 2014). "Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy". The Journal of Cell Biology. 207 (1): 139–158. doi:10.1083/jcb.201402079. PMC 4195829. PMID 25313409.
^ Damsker JM, Conklin LS, Sadri S, Dillingham BC, Panchapakesan K, Heier CR, et al. (September 2016). "VBP15, a novel dissociative steroid compound, reduces NFκB-induced expression of inflammatory cytokines in vitro and symptoms of murine trinitrobenzene sulfonic acid-induced colitis". Inflammation Research. 65 (9): 737–743. doi:10.1007/s00011-016-0956-8. PMID 27261270. S2CID 18698831.
^ Heier CR, Yu Q, Fiorillo AA, Tully CB, Tucker A, Mazala DA, et al. (February 2019). "Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy". Life Sci Alliance. 2 (1): e201800186. doi:10.26508/lsa.201800186. PMC 6371196. PMID 30745312.
^ ^a ^b "Drug Trials Snapshots: Agamree". U.S. Food and Drug Administration (FDA). 16 February 2024. Retrieved 14 March 2024. This article incorporates text from this source, which is in the public domain.
^ Cite error: The named reference FDA approval was invoked but never defined (see the help page).

[Agamree_FDA_label-1] "Agamree- vamorolone kit". DailyMed. 26 October 2023. Retrieved 20 November 2023.

[Agamree_EPAR-2] Cite error: The named reference Agamree EPAR was invoked but never defined (see the help page).

[Agamree_Product_information-3] "Agamree Product information". Union Register of medicinal products. 15 December 2023. Retrieved 26 December 2023.

[AdisInsight-4] "Vamorolone - ReveraGen Biopharma". AdisInsight. Springer Nature Switzerland AG. Archived from the original on 7 October 2017. Retrieved 2 July 2017.

[5] Reeves EK, Hoffman EP, Nagaraju K, Damsker JM, McCall JM (April 2013). "VBP15: preclinical characterization of a novel anti-inflammatory delta 9,11 steroid". Bioorganic & Medicinal Chemistry. 21 (8): 2241–2249. doi:10.1016/j.bmc.2013.02.009. PMC 4088988. PMID 23498916.

[6] Heier CR, Damsker JM, Yu Q, Dillingham BC, Huynh T, Van der Meulen JH, et al. (October 2013). "VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects". EMBO Molecular Medicine. 5 (10): 1569–1585. doi:10.1002/emmm.201302621. PMC 3799580. PMID 24014378.

[7] Dadgar S, Wang Z, Johnston H, Kesari A, Nagaraju K, Chen YW, et al. (October 2014). "Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy". The Journal of Cell Biology. 207 (1): 139–158. doi:10.1083/jcb.201402079. PMC 4195829. PMID 25313409.

[8] Damsker JM, Conklin LS, Sadri S, Dillingham BC, Panchapakesan K, Heier CR, et al. (September 2016). "VBP15, a novel dissociative steroid compound, reduces NFκB-induced expression of inflammatory cytokines in vitro and symptoms of murine trinitrobenzene sulfonic acid-induced colitis". Inflammation Research. 65 (9): 737–743. doi:10.1007/s00011-016-0956-8. PMID 27261270. S2CID 18698831.

[pmid30745312-9] Heier CR, Yu Q, Fiorillo AA, Tully CB, Tucker A, Mazala DA, et al. (February 2019). "Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy". Life Sci Alliance. 2 (1): e201800186. doi:10.26508/lsa.201800186. PMC 6371196. PMID 30745312.

[Agamree_FDA_snapshot-10] "Drug Trials Snapshots: Agamree". U.S. Food and Drug Administration (FDA). 16 February 2024. Retrieved 14 March 2024. This article incorporates text from this source, which is in the public domain.

[FDA_approval-11] Cite error: The named reference FDA approval was invoked but never defined (see the help page).

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