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

Stem definitionDrug idCAS RN
antisense oligonucleotides 5733 2088232-70-4

Description:

MoleculeDescription

Molfile Inchi Smiles

Synonyms:

  • tofersen
  • tofersen sodium
  • qalsody
  • BIIB067
Tofersen is an antisense oligonucleotide that causes degradation of SOD1 mRNA through binding to SOD1 mRNA, which results in a reduction of SOD1 protein synthesis.
  • Molecular weight: 7127.85
  • Formula: C230H317N72O123P19S15
  • CLOGP:
  • LIPINSKI: 3
  • HAC: 195
  • HDO: 29
  • TPSA: 2429.18
  • ALOGS:
  • ROTB: 156

Drug dosage:

None

ADMET properties:

None

Predicted pharmacokinetic/toxicological properties (PK-AZ):

None

Approvals:

DateAgencyCompanyOrphan
April 25, 2023 FDA BIOGEN MA

FDA Adverse Event Reporting System (Female)

None

FDA Adverse Event Reporting System (Male)

None

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 86.96 62.92 11 145 1086 110588057
CSF protein increased 83.58 62.92 11 145 1480 110587663

FDA Adverse Event Reporting System (Pediatric)

None

Pharmacologic Action:

SourceCodeDescription
ATC N07XX22 NERVOUS SYSTEM
OTHER NERVOUS SYSTEM DRUGS
OTHER NERVOUS SYSTEM DRUGS
Other nervous system drugs
FDA CS M0025055 Oligonucleotides, Antisense
FDA EPC N0000191626 Antisense Oligonucleotide

Related Drugs by ATC class:

N07XX Other nervous system drugs 23 drugs
amifampridinearimoclomoldeutetrabenazinedextromethorphanedaravoneeplontersenfampridineinotersen laquinimodlevacetylleucineomaveloxolonepatisiran show all 23 →

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

DiseaseRelationSNOMED_IDDOID
Amyotrophic lateral sclerosis indication 86044005 DOID:332




🐶 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
100MG/15ML (6.7MG/ML) QALSODY BIOGEN MA N215887 April 25, 2023 RX SOLUTION INTRATHECAL 10669546 April 1, 2035 TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) IN ADULTS WHO HAVE A MUTATION IN THE SUPEROXIDE DISMUTASE 1 (SOD1) GENE
100MG/15ML (6.7MG/ML) QALSODY BIOGEN MA N215887 April 25, 2023 RX SOLUTION INTRATHECAL 10968453 April 1, 2035 TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) IN ADULTS WHO HAVE A MUTATION IN THE SUPEROXIDE DISMUTASE 1 (SOD1) GENE

Orange Book exclusivity data (new drug applications)

Formulation strengthTrade nameApplicantApplication numberApproval dateTypeDose formRouteExclusivity dateDescription
100MG/15ML (6.7MG/ML) QALSODY BIOGEN MA N215887 April 25, 2023 RX SOLUTION INTRATHECAL April 25, 2028 NEW CHEMICAL ENTITY
100MG/15ML (6.7MG/ML) QALSODY BIOGEN MA N215887 April 25, 2023 RX SOLUTION INTRATHECAL April 25, 2030 TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) IN ADULTS WHO HAVE A MUTATION IN THE SUPEROXIDE DISMUTASE 1 (SOD1) GENE

Bioactivity Summary:

TargetClassPharosUniProtActionTypeActivity value
(-log[M])		Mechanism
action		Bioact sourceMoA source
Superoxide dismutase 1 (SOD1) mRNA RNA BINDING AGENT UNKNOWN DRUG LABEL

External reference:

IDSource
CHEBI:755937 CHEBI
CHEMBL3833346 ChEMBL_ID
C000709090 MESH_SUPPLEMENTAL_RECORD_UI
13536 IUPHAR_LIGAND_ID
DB14782 DRUGBANK_ID
44941311000001100 SNOMEDCT_US
4042212 VANDF
C5139925 UMLSCUI
CHEMBL4297234 ChEMBL_ID
10804 INN_ID
1898254-60-8 SECONDARY_CAS_RN
366714 MMSL
41435 MMSL
d10039 MMSL
019401 NDDF
D11811 KEGG_DRUG
2NU6F9601K UNII
2634995 RXNORM

Pharmaceutical products:

ProductCategoryIngredientsNDCFormQuantityRouteMarketingLabel
QALSODY HUMAN PRESCRIPTION DRUG LABEL 1 64406-109 INJECTION 100 mg INTRATHECAL NDA 25 sections