All FDA-approved EMA-approved PMDA-approved
Target Card Uniprot Example: P23975

nusinersen 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | PK/Tox | Related Drugs | 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

Drug dosage:

Dose	Unit	Route
0.10	mg	P

ADMET properties:

None

Predicted pharmacokinetic/toxicological properties (PK-AZ):

None

Approvals:

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

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	347.97	38.50	52	2374	845	90625176
Scoliosis	207.35	38.50	48	2378	8859	90617162
Rhinovirus infection	148.68	38.50	34	2392	5929	90620092
Protein urine present	92.82	38.50	24	2402	6960	90619061
Respiratory syncytial virus infection	78.74	38.50	23	2403	10457	90615564
Cerebrospinal fluid leakage	73.28	38.50	15	2411	1552	90624469
Acute respiratory failure	65.94	38.50	29	2397	42889	90583132
Death	65.09	38.50	70	2356	456481	90169540
Respiratory disorder	60.28	38.50	31	2395	64785	90561236
Traumatic lumbar puncture	58.69	38.50	7	2419	15	90626006
Cardio-respiratory arrest	55.28	38.50	29	2397	63111	90562910
Procedural pain	52.71	38.50	23	2403	33337	90592684
Respiratory failure	52.44	38.50	35	2391	118383	90507638
Pyrexia	48.84	38.50	73	2353	660412	89965609
Respiratory tract infection	45.71	38.50	25	2401	59053	90566968
Cerebrospinal fluid reservoir placement	45.30	38.50	5	2421	3	90626018
Cardiac arrest	44.01	38.50	30	2396	104905	90521116
Pneumonia	43.57	38.50	68	2358	637991	89988030
Meningitis chemical	40.58	38.50	7	2419	286	90625735
Pneumonia aspiration	39.25	38.50	20	2406	40920	90585101

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	363.54	34.69	51	2471	206	42618607
Scoliosis	180.96	34.69	41	2481	3085	42615728
Intracranial pressure increased	83.51	34.69	24	2498	4650	42614163
Respiratory tract infection	82.02	34.69	37	2485	26285	42592528
Protein urine present	74.16	34.69	23	2499	5778	42613035
Respiratory failure	66.06	34.69	57	2465	128075	42490738
Respiratory disorder	65.48	34.69	30	2492	22055	42596758
Cerebrospinal fluid leakage	55.79	34.69	12	2510	706	42618107
Procedural pain	53.75	34.69	18	2504	5761	42613052
Pneumonia	51.19	34.69	90	2432	422059	42196754
Rhinovirus infection	43.97	34.69	16	2506	6532	42612281
Respiratory syncytial virus infection	42.59	34.69	16	2506	7136	42611677
Respiratory distress	42.54	34.69	28	2494	41792	42577021
Respiratory arrest	41.58	34.69	24	2498	28400	42590413
Procedural vomiting	36.60	34.69	7	2515	227	42618586
Chronic respiratory failure	35.99	34.69	10	2512	1712	42617101
Cardiac arrest	35.95	34.69	38	2484	109282	42509531
Therapeutic response shortened	35.77	34.69	17	2505	13537	42605276
CSF pressure increased	34.72	34.69	5	2517	25	42618788

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	607.04	33.02	90	3885	1007	110584317
Scoliosis	194.04	33.02	48	3927	8685	110576639
Rhinovirus infection	142.52	33.02	40	3935	11749	110573575
Protein urine present	125.43	33.02	36	3939	11448	110573876
Cerebrospinal fluid leakage	113.89	33.02	24	3951	2124	110583200
Respiratory tract infection	95.27	33.02	48	3927	71569	110513755
Traumatic lumbar puncture	83.11	33.02	9	3966	0	110585324
Respiratory failure	79.34	33.02	64	3911	215268	110370056
Respiratory syncytial virus infection	72.39	33.02	25	3950	14459	110570865
Pneumonia	68.93	33.02	117	3858	876597	109708727
Procedural pain	66.05	33.02	30	3945	35535	110549789
Respiratory arrest	65.78	33.02	35	3940	58264	110527060
Pyrexia	61.68	33.02	113	3862	898152	109687172
Death	58.47	33.02	96	3879	698773	109886551
Procedural vomiting	57.55	33.02	12	3963	1008	110584316
Pneumonia aspiration	57.49	33.02	36	3939	81197	110504127
Cardiac arrest	57.08	33.02	51	3924	196702	110388622
Atelectasis	55.90	33.02	27	3948	36685	110548639
Chronic respiratory failure	55.25	33.02	15	3960	3888	110581436
Respiratory distress	55.06	33.02	33	3942	68841	110516483
Acute respiratory failure	54.89	33.02	34	3941	75226	110510098
Cardio-respiratory arrest	52.57	33.02	39	3936	115797	110469527
Aspartate aminotransferase increased	47.44	33.02	42	3933	159894	110425430
Respiratory disorder	46.43	33.02	30	3945	71352	110513972
Enterovirus infection	45.62	33.02	12	3963	2758	110582566
Cerebrospinal fluid reservoir placement	42.23	33.02	5	3970	6	110585318
Aspiration	39.20	33.02	20	3955	30513	110554811
Alanine aminotransferase increased	38.79	33.02	40	3935	183733	110401591
Troponin I increased	37.59	33.02	12	3963	5440	110579884
CSF protein increased	37.18	33.02	9	3966	1482	110583842

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

Related Drugs by ATC class:

M09AX Other drugs for disorders of the musculo-skeletal system 11 drugs

aceneuramic acidatalurencasimerseneteplirsengivinostatgolodirsenhyaluronic acidonasemnogene abeparvovecpalovarotenerisdiplamviltolarsen

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	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	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	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	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	9717750	June 17, 2030	TREATMENT OF TYPE II SPINAL MUSCULAR ATROPHY
EQ 28MG BASE/5ML (EQ 5.6MG BASE/ML)	SPINRAZA	BIOGEN	N209531	March 27, 2026	RX	SOLUTION	INTRATHECAL	9717750	June 17, 2030	TREATMENT OF SPINAL MUSCULAR ATROPHY
EQ 28MG BASE/5ML (EQ 5.6MG BASE/ML)	SPINRAZA	BIOGEN	N209531	March 27, 2026	RX	SOLUTION	INTRATHECAL	9717750	June 17, 2030	TREATMENT OF SPINAL MUSCULAR ATROPHY BY INCREASING EXON-7 INCLUSION IN SMN2 MRNA
EQ 28MG BASE/5ML (EQ 5.6MG BASE/ML)	SPINRAZA	BIOGEN	N209531	March 27, 2026	RX	SOLUTION	INTRATHECAL	9717750	June 17, 2030	TREATMENT OF TYPE III SPINAL MUSCULAR ATROPHY
EQ 28MG BASE/5ML (EQ 5.6MG BASE/ML)	SPINRAZA	BIOGEN	N209531	March 27, 2026	RX	SOLUTION	INTRATHECAL	9717750	June 17, 2030	TREATMENT OF TYPE II SPINAL MUSCULAR ATROPHY
EQ 50MG BASE/5ML (EQ 10MG BASE/ML)	SPINRAZA	BIOGEN	N209531	March 27, 2026	RX	SOLUTION	INTRATHECAL	9717750	June 17, 2030	TREATMENT OF SPINAL MUSCULAR ATROPHY
EQ 50MG BASE/5ML (EQ 10MG BASE/ML)	SPINRAZA	BIOGEN	N209531	March 27, 2026	RX	SOLUTION	INTRATHECAL	9717750	June 17, 2030	TREATMENT OF SPINAL MUSCULAR ATROPHY BY INCREASING EXON-7 INCLUSION IN SMN2 MRNA
EQ 50MG BASE/5ML (EQ 10MG BASE/ML)	SPINRAZA	BIOGEN	N209531	March 27, 2026	RX	SOLUTION	INTRATHECAL	9717750	June 17, 2030	TREATMENT OF TYPE III SPINAL MUSCULAR ATROPHY
EQ 50MG BASE/5ML (EQ 10MG BASE/ML)	SPINRAZA	BIOGEN	N209531	March 27, 2026	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	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	8980853	Nov. 24, 2030	TREATMENT OF INFANTILE-ONSET SPINAL MUSCULAR ATROPHY
EQ 28MG BASE/5ML (EQ 5.6MG BASE/ML)	SPINRAZA	BIOGEN	N209531	March 27, 2026	RX	SOLUTION	INTRATHECAL	8980853	Nov. 24, 2030	TREATMENT OF INFANTILE-ONSET SPINAL MUSCULAR ATROPHY
EQ 50MG BASE/5ML (EQ 10MG BASE/ML)	SPINRAZA	BIOGEN	N209531	March 27, 2026	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	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	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	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	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	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	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	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	10436802	Sept. 11, 2035	TREATMENT OF TYPE II SPINAL MUSCULAR ATROPHY
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	12013403	March 4, 2036	TREATMENT OF INFANTILE-ONSET SPINAL MUSCULAR ATROPHY
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	12013403	March 4, 2036	TREATMENT OF SPINAL MUSCULAR ATROPHY
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	12013403	March 4, 2036	TREATMENT OF SPINAL MUSCULAR ATROPHY BY INCREASING EXON-7 INCLUSION IN SMN2 MRNA
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	12013403	March 4, 2036	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	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	12013403	March 4, 2036	TREATMENT OF TYPE III SPINAL MUSCULAR ATROPHY
EQ 12MG BASE/5ML (EQ 2.4MG BASE/ML)	SPINRAZA	BIOGEN	N209531	Dec. 23, 2016	RX	SOLUTION	INTRATHECAL	12013403	March 4, 2036	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 28MG BASE/5ML (EQ 5.6MG BASE/ML)	SPINRAZA	BIOGEN	N209531	March 27, 2026	RX	SOLUTION	INTRATHECAL	March 27, 2029	NEW STRENGTH
EQ 50MG BASE/5ML (EQ 10MG BASE/ML)	SPINRAZA	BIOGEN	N209531	March 27, 2026	RX	SOLUTION	INTRATHECAL	March 27, 2029	NEW STRENGTH

Bioactivity Summary:

Target	Class	Pharos	UniProt	Action	Type	Activity value (-log[M])	Mechanism action	Bioact source	MoA source
Survival motor neuron protein mRNA	RNA	Q16637	SMN_HUMAN	ANTISENSE INHIBITOR				UNKNOWN	DRUG LABEL

External reference:

ID	Source
5Z9SP3X666	UNII
4036321	VANDF
C4301236	UMLSCUI
CHEBI:755256	CHEBI
CHEMBL3301572	ChEMBL_ID
DB13161	DRUGBANK_ID
D10791	KEGG_DRUG
CHEMBL3833342	ChEMBL_ID
10003	INN_ID
C000590926	MESH_SUPPLEMENTAL_RECORD_UI
9416	IUPHAR_LIGAND_ID
1155774000	SNOMEDCT_US
723985005	SNOMEDCT_US
763556007	SNOMEDCT_US
248506	MMSL
32201	MMSL
33098	MMSL
d08511	MMSL
131801471	PUBCHEM_CID
017062	NDDF
017065	NDDF
1863556	RXNORM

Pharmaceutical products:

Product	Category	Ingredients	NDC	Form	Quantity	Route	Marketing	Label
Spinraza	HUMAN PRESCRIPTION DRUG LABEL	1	64406-036	INJECTION, SOLUTION	5.60 mg	INTRATHECAL	NDA	28 sections
Spinraza	HUMAN PRESCRIPTION DRUG LABEL	1	64406-037	INJECTION, SOLUTION	10 mg	INTRATHECAL	NDA	28 sections
Spinraza	HUMAN PRESCRIPTION DRUG LABEL	1	64406-058	INJECTION, SOLUTION	2.40 mg	INTRATHECAL	NDA	28 sections