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

Stem definitionDrug idCAS RN
antisense oligonucleotides 5207 1258984-36-9

Description:

MoleculeDescription

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:

DoseUnitRoute
0.10 mg P

ADMET properties:

None

Approvals:

DateAgencyCompanyOrphan
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 termLikelihood ratioLikelihood ratio thresholdPatients taking drug having adverse eventPatients taking drug not having adverse eventPatients not taking drug having adverse eventPatients 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 termLikelihood ratioLikelihood ratio thresholdPatients taking drug having adverse eventPatients taking drug not having adverse eventPatients not taking drug having adverse eventPatients 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 termLikelihood ratioLikelihood ratio thresholdPatients taking drug having adverse eventPatients taking drug not having adverse eventPatients not taking drug having adverse eventPatients 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:

SourceCodeDescription
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|

DiseaseRelationSNOMED_IDDOID
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 strengthTrade nameApplicantApplication numberApproval dateTypeDose formRoutePatent numberPatent expiration datePatent 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 strengthTrade nameApplicantApplication numberApproval dateTypeDose formRouteExclusivity dateDescription
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:

IDSource
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:

ProductCategoryIngredientsNDCFormQuantityRouteMarketingLabel
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