onasemnogene abeparvovec 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | BioActivity |

Stem definition	Drug id	CAS RN
	5332	None

Description:

Molecule	Description
Synonyms: onasemnogene abeparvovec onasemnogene abeparvovec-xioi zolgensma AVXS-101	ZOLGENSMA is a recombinant AAV9-based gene therapy designed to deliver a copy of the gene encoding the human SMN protein. SMA is caused by a bi-allelic mutation in the SMN1 gene, which results in insufficient SMN protein expression. Intravenous administration of ZOLGENSMA that results in cell transduction and expression of the SMN protein has been observed in two human case studies Molecular weight: Formula: CLOGP: LIPINSKI: None HAC: None HDO: None TPSA: ALOGS: ROTB: None

Molecule

Description

Synonyms:

onasemnogene abeparvovec
onasemnogene abeparvovec-xioi
zolgensma
AVXS-101

ZOLGENSMA is a recombinant AAV9-based gene therapy designed to deliver a copy of the gene encoding the human SMN protein. SMA is caused by a bi-allelic mutation in the SMN1 gene, which results in insufficient SMN protein expression. Intravenous administration of ZOLGENSMA that results in cell transduction and expression of the SMN protein has been observed in two human case studies

Molecular weight:
Formula:
CLOGP:
LIPINSKI: None
HAC: None
HDO: None
TPSA:
ALOGS:
ROTB: None

Drug dosage:

None

ADMET properties:

None

Approvals:

Date	Agency	Company	Orphan
May 18, 2020	EMA	Novartis Europharm Limited
May 24, 2019	FDA	NOVARTIS PHARMS CORP

FDA Adverse Event Reporting System (Female)

MedDRA adverse event term	Likelihood ratio	Likelihood ratio threshold	Patients taking drug having adverse event	Patients taking drug not having adverse event	Patients not taking drug having adverse event	Patients not taking drug not having adverse event
Aspartate aminotransferase increased	296.92	50.12	112	2043	90165	63396702
Alanine aminotransferase increased	253.98	50.12	104	2051	103666	63383201
Troponin I increased	180.01	50.12	35	2120	2203	63484664
Pyrexia	168.15	50.12	133	2022	470345	63016522
Thrombocytopenia	148.98	50.12	81	2074	151076	63335791
Transaminases increased	125.00	50.12	45	2110	31322	63455545
Vomiting	97.03	50.12	106	2049	559511	62927356
Thrombotic microangiopathy	81.62	50.12	25	2130	10536	63476331
Platelet count decreased	71.38	50.12	46	2109	116076	63370791
Troponin T increased	67.23	50.12	14	2141	1240	63485627
Blood lactate dehydrogenase increased	62.46	50.12	25	2130	23091	63463776
Hepatic enzyme increased	56.56	50.12	50	2105	202278	63284589

FDA Adverse Event Reporting System (Male)

MedDRA adverse event term	Likelihood ratio	Likelihood ratio threshold	Patients taking drug having adverse event	Patients taking drug not having adverse event	Patients not taking drug having adverse event	Patients not taking drug not having adverse event
Aspartate aminotransferase increased	235.50	47.88	95	1511	67688	34887637
Alanine aminotransferase increased	193.32	47.88	87	1519	80728	34874597
Troponin I increased	191.93	47.88	40	1566	2642	34952683
Pyrexia	133.38	47.88	114	1492	332899	34622426
Hepatic enzyme increased	115.52	47.88	49	1557	39031	34916294
Transaminases increased	87.10	47.88	36	1570	26787	34928538
Thrombocytopenia	75.46	47.88	60	1546	156187	34799138
Vomiting	75.29	47.88	73	1533	247548	34707777
Liver function test increased	66.37	47.88	25	1581	14540	34940785
Rhinovirus infection	54.59	47.88	16	1590	4283	34951042
Platelet count decreased	49.30	47.88	42	1564	119675	34835650
Blood lactate dehydrogenase increased	49.07	47.88	23	1583	22967	34932358

FDA Adverse Event Reporting System (Geriatric)

MedDRA adverse event term	Likelihood ratio	Likelihood ratio threshold	Patients taking drug having adverse event	Patients taking drug not having adverse event	Patients not taking drug having adverse event	Patients not taking drug not having adverse event
Aspartate aminotransferase increased	442.64	46.39	176	3015	138465	79602732
Alanine aminotransferase increased	365.93	46.39	161	3030	162409	79578788
Pyrexia	213.04	46.39	191	3000	678518	79062679
Troponin I increased	207.13	46.39	46	3145	4692	79736505
Thrombocytopenia	198.47	46.39	126	3065	265133	79476064
Transaminases increased	182.80	46.39	71	3120	51672	79689525
Hepatic enzyme increased	124.46	46.39	82	3109	182528	79558669
Vomiting	114.25	46.39	136	3055	665692	79075505
Platelet count decreased	107.96	46.39	77	3114	194587	79546610
Blood lactate dehydrogenase increased	107.94	46.39	45	3146	39125	79702072
Thrombotic microangiopathy	83.05	46.39	31	3160	20138	79721059
Liver function test increased	62.78	46.39	31	3160	39742	79701455
Rhinovirus infection	59.07	46.39	19	3172	7934	79733263
Troponin increased	52.58	46.39	22	3169	19233	79721964
Troponin T increased	50.55	46.39	14	3177	3508	79737689
Increased bronchial secretion	46.72	46.39	13	3178	3314	79737883

FDA Adverse Event Reporting System (Pediatric)

None

Pharmacologic Action:

Source	Code	Description
ATC	M09AX09	MUSCULO-SKELETAL SYSTEM OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM Other drugs for disorders of the musculo-skeletal system

Drug Use | Suggest Off label Use Form| |View source of the data|

Disease	Relation	SNOMED_ID	DOID
Spinal muscular atrophy	indication	5262007	DOID:12377

🐶 Veterinary Drug Use

None

🐶 Veterinary products

None

Acid dissociation constants calculated using MoKa v3.0.0

None

Orange Book patent data (new drug applications)

None

Orange Book exclusivity data (new drug applications)

None

Bioactivity Summary:

None

External reference:

ID	Source
MLU3LU3EVV	UNII
C5140028	UMLSCUI
CHEMBL4297240	ChEMBL_ID
DB15528	DRUGBANK_ID
D11559	KEGG_DRUG
10619	INN_ID
C000710948	MESH_SUPPLEMENTAL_RECORD_UI
018004	NDDF
788110002	SNOMEDCT_US
789426004	SNOMEDCT_US
1922968-73-7	SECONDARY_CAS_RN
2170225	RXNORM
36975	MMSL
36979	MMSL
d09287	MMSL

Pharmaceutical products:

None