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

Stem definition	Drug id	CAS RN
antisense oligonucleotides	5207	1258984-36-9

Description:

Molecule	Description
Synonyms: nusinersen spinraza ISIS 396443 nusinersen sodium	Nusinersen is a modified antisense oligonucleotide, where the 2' hydroxy groups of the ribofuranosyl rings are replaced with 2'-O-2-methoxyethyl groups and the phosphate linkages are replaced with phosphorothioate linkages. Nusinersen binds to a specific sequence in the intron downstream of exon 7 of the SMN2 transcript. Nusinersen is designed to treat spinal muscular atrophy (SMA) caused by mutations in chromosome 5q that lead to SMN protein deficiency. Using in vitro assays and studies in transgenic animal models of SMA, SPINRAZA was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein. Molecular weight: 7500.86 Formula: C234H323N61Na17O128P17S17 CLOGP: LIPINSKI: None HAC: None HDO: None TPSA: 2257.90 ALOGS: ROTB: None

Molecule

Description

Synonyms:

nusinersen
spinraza
ISIS 396443
nusinersen sodium

Nusinersen is a modified antisense oligonucleotide, where the 2' hydroxy groups of the ribofuranosyl rings are replaced with 2'-O-2-methoxyethyl groups and the phosphate linkages are replaced with phosphorothioate linkages. Nusinersen binds to a specific sequence in the intron downstream of exon 7 of the SMN2 transcript. Nusinersen is designed to treat spinal muscular atrophy (SMA) caused by mutations in chromosome 5q that lead to SMN protein deficiency. Using in vitro assays and studies in transgenic animal models of SMA, SPINRAZA was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein.

Molecular weight: 7500.86
Formula: C234H323N61Na17O128P17S17
CLOGP:
LIPINSKI: None
HAC: None
HDO: None
TPSA: 2257.90
ALOGS:
ROTB: None

Drug dosage:

Dose	Unit	Route
0.10	mg	P

ADMET properties:

None

Approvals:

Date	Agency	Company
May 30, 2017	EMA	Biogen Idec Ltd
Dec. 23, 2016	FDA	BIOGEN
July 3, 2017	PMDA	Biogen Japan Ltd

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
Post lumbar puncture syndrome	282.18	39.20	42	1904	574	63486502
Scoliosis	159.88	39.20	39	1907	7849	63479227
Rhinovirus infection	137.05	39.20	31	1915	4498	63482578
Protein urine present	87.93	39.20	23	1923	6097	63480979
Respiratory syncytial virus infection	75.53	39.20	21	1925	6997	63480079
Cerebrospinal fluid leakage	69.39	39.20	14	1932	1176	63485900
Acute respiratory failure	58.49	39.20	26	1920	34459	63452617
Respiratory disorder	52.30	39.20	25	1921	39057	63448019
Pyrexia	49.50	39.20	66	1880	470412	63016664
Respiratory tract infection	47.91	39.20	24	1922	41459	63445617
Cerebrospinal fluid reservoir placement	46.97	39.20	5	1941	0	63487076
Traumatic lumbar puncture	46.97	39.20	5	1941	0	63487076
Respiratory failure	45.81	39.20	32	1914	101826	63385250
Cardio-respiratory arrest	44.88	39.20	26	1920	59933	63427143
Procedural pain	42.21	39.20	18	1928	21551	63465525

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
Post lumbar puncture syndrome	300.51	39.96	42	1897	178	34954814
Scoliosis	117.87	39.96	28	1911	2765	34952227
Respiratory tract infection	77.39	39.96	32	1907	19680	34935312
Protein urine present	73.65	39.96	22	1917	5206	34949786
Intracranial pressure increased	70.58	39.96	20	1919	3935	34951057
Respiratory disorder	68	39.96	29	1910	19235	34935757
Respiratory failure	53.97	39.96	46	1893	108526	34846466
Rhinovirus infection	51.60	39.96	16	1923	4283	34950709
Pneumonia	47.33	39.96	77	1862	362550	34592442
Respiratory distress	45.71	39.96	27	1912	35638	34919354
Cerebrospinal fluid leakage	42.47	39.96	9	1930	527	34954465
Respiratory syncytial virus infection	42.05	39.96	14	1925	4695	34950297

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
Post lumbar puncture syndrome	520.65	35.25	76	3049	689	79740574
Rhinovirus infection	145.07	35.25	38	3087	7915	79733348
Scoliosis	123.42	35.25	33	3092	7431	79733832
Protein urine present	122.42	35.25	35	3090	10077	79731186
Respiratory tract infection	102.62	35.25	46	3079	48643	79692620
Cerebrospinal fluid leakage	94.23	35.25	20	3105	1679	79739584
Respiratory failure	73.54	35.25	59	3066	180852	79560411
Respiratory syncytial virus infection	68.41	35.25	22	3103	9380	79731883
Pneumonia	60.59	35.25	99	3026	660147	79081116
Traumatic lumbar puncture	54.89	35.25	6	3119	0	79741263
Atelectasis	53.98	35.25	26	3099	32231	79709032
Procedural pain	52.41	35.25	23	3102	23061	79718202
Respiratory distress	52.24	35.25	31	3094	58308	79682955
Pyrexia	52.01	35.25	94	3031	678615	79062648
Pneumonia aspiration	50.79	35.25	32	3093	66935	79674328
Cardio-respiratory arrest	49.66	35.25	38	3087	108472	79632791
Chronic respiratory failure	48.23	35.25	13	3112	3009	79738254
Respiratory arrest	47.44	35.25	29	3096	57521	79683742
Respiratory disorder	45.90	35.25	26	3099	44830	79696433
Cerebrospinal fluid reservoir placement	45.74	35.25	5	3120	0	79741263
Enterovirus infection	43.78	35.25	11	3114	1936	79739327
Aspiration	42.27	35.25	19	3106	20139	79721124
Acute respiratory failure	41.54	35.25	27	3098	59514	79681749

FDA Adverse Event Reporting System (Pediatric)

None

Pharmacologic Action:

Source	Code	Description
ATC	M09AX07	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
FDA CS	M0025055	Oligonucleotides, Antisense
FDA PE	N0000009533	Increased Protein Synthesis
FDA EPC	N0000191626	Antisense Oligonucleotide
FDA EPC	N0000193222	Survival Motor Neuron-2-directed RNA Interaction

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)

Formulation strength	Trade name	Applicant	Application number	Approval date	Type	Dose form	Route	Patent number	Patent expiration date	Patent use
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN IDEC	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	10266822	Dec. 5, 2025	TREATMENT OF SPINAL MUSCULAR ATROPHY
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN IDEC	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	10266822	Dec. 5, 2025	TREATMENT OF SPINAL MUSCULAR ATROPHY BY INCREASING EXON-7 INCLUSION IN SMN2 MRNA
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN IDEC	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	10266822	Dec. 5, 2025	TREATMENT OF SPINAL MUSCULAR ATROPHY BY INHIBITING AN SMN2 PRE-MRNA INTRONIC SPLICING SILENCER SITE
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN IDEC	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	8110560	Dec. 5, 2025	TREATMENT OF SPINAL MUSCULAR ATROPHY
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN IDEC	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	8110560	Dec. 5, 2025	TREATMENT OF SPINAL MUSCULAR ATROPHY BY INCREASING EXON-7 INCLUSION IN SMN2 MRNA
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN IDEC	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	8110560	Dec. 5, 2025	TREATMENT OF SPINAL MUSCULAR ATROPHY BY INHIBITING AN SMN2 PRE-MRNA INTRONIC SPLICING SILENCER SITE
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN IDEC	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	9717750	June 17, 2030	TREATMENT OF SPINAL MUSCULAR ATROPHY
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN IDEC	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	9717750	June 17, 2030	TREATMENT OF SPINAL MUSCULAR ATROPHY BY INCREASING EXON-7 INCLUSION IN SMN2 MRNA
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN IDEC	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	9717750	June 17, 2030	TREATMENT OF TYPE III SPINAL MUSCULAR ATROPHY
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN IDEC	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	9717750	June 17, 2030	TREATMENT OF TYPE II SPINAL MUSCULAR ATROPHY
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN IDEC	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	8980853	Nov. 24, 2030	TREATMENT OF INFANTILE-ONSET SPINAL MUSCULAR ATROPHY
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN IDEC	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	9926559	Jan. 9, 2034	TREATMENT OF SPINAL MUSCULAR ATROPHY
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN IDEC	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	10436802	Sept. 11, 2035	TREATMENT OF INFANTILE-ONSET SPINAL MUSCULAR ATROPHY
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN IDEC	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	10436802	Sept. 11, 2035	TREATMENT OF SPINAL MUSCULAR ATROPHY
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN IDEC	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	10436802	Sept. 11, 2035	TREATMENT OF SPINAL MUSCULAR ATROPHY BY INCREASING EXON-7 INCLUSION IN SMN2 MRNA
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN IDEC	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	10436802	Sept. 11, 2035	TREATMENT OF SPINAL MUSCULAR ATROPHY BY INHIBITING AN SMN2 PRE-MRNA INTRONIC SPLICING SILENCER SITE
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN IDEC	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	10436802	Sept. 11, 2035	TREATMENT OF TYPE III SPINAL MUSCULAR ATROPHY
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN IDEC	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	10436802	Sept. 11, 2035	TREATMENT OF TYPE II SPINAL MUSCULAR ATROPHY

Orange Book exclusivity data (new drug applications)

Formulation strength	Trade name	Applicant	Application number	Approval date	Type	Dose form	Route	Exclusivity date	Description
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN IDEC	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	Dec. 23, 2023	TREATMENT OF SPINAL MUSCULAR ATROPHY IN PEDIATRIC AND ADULT PATIENTS

Bioactivity Summary:

None

External reference:

ID	Source
5Z9SP3X666	UNII
4036321	VANDF
C4301236	UMLSCUI
CHEMBL3301572	ChEMBL_ID
131801471	PUBCHEM_CID
DB13161	DRUGBANK_ID
D10791	KEGG_DRUG
CHEMBL3833342	ChEMBL_ID
10003	INN_ID
9416	IUPHAR_LIGAND_ID
248506	MMSL
32201	MMSL
33098	MMSL
d08511	MMSL
017062	NDDF
017065	NDDF
1155774000	SNOMEDCT_US
723985005	SNOMEDCT_US
763556007	SNOMEDCT_US
1863556	RXNORM
C000590926	MESH_SUPPLEMENTAL_RECORD_UI

Pharmaceutical products:

Product	Category	Ingredients	NDC	Form	Quantity	Route	Marketing	Label
Spinraza	HUMAN PRESCRIPTION DRUG LABEL	1	64406-058	INJECTION, SOLUTION	2.40 mg	INTRATHECAL	NDA	26 sections
Spinraza	HUMAN PRESCRIPTION DRUG LABEL	1	64406-058	INJECTION, SOLUTION	2.40 mg	INTRATHECAL	NDA	26 sections