Onasemnogene abeparvovec
Highest phase reached
Approved
Drug type
Gene
First approval year
2020
- First approval year
- 2020
- Listing at source
- Listed
- Highest phase reached
- Approved
- Drug type
- Gene
- Drug name
- Onasemnogene abeparvovec
- zolgensma
- Zolgensma
- 10 mg BGM0504 injection administered subcutaneously (SC) once a week
- 10 mg bgm0504 injection administered subcutaneously sc once a week
- 140 mg SC
- 210 mg SC
- 280 mg SC
- 350 mg SC
- 70 mg SC
- Adalimumab 40 mg subcutaneous (SC) every other week (EOW)
- avxs 101
- AVXS-101
- AZD9793 Subcutaneous (SC) monotherapy
- CM313(SC) injection-high dose
- CM313(SC) injection-low dose
- Daratumumab Subcutaneous (SC) Administration
- ePGT121v1-LS (SC)
- GB-0895 (SC)
- GI-102 subcutaneous (SC)
- Guselkumab (SC): Dose 1
- Guselkumab (SC): Dose 2
- LY3200327 (SC)
- MER511 (SC)
- MER511 (SC) for MAD
- MK-2118 (SC)
- Modified Live Tetravalent Chimeric Dengue Vaccine (SC)
- non replicating recombinant self complementary sc adeno associated virus serotype 9 aav9 vector containing the cdna of the survival of motor neuron 2 smn2 gene under the control of the hybrid cytomegalovirus cmv enhancer chicken beta actin promoter cba
And 31 more, listed on the interactive page.
- ATC classification codes
- M09AX09
Sources for recorded values
- CAS registry numbers
- 1922968-73-7
- ChEMBL ID
- CHEMBL4297240
- RxNorm IDs (RxCUI)
- 2170226
- FDA ingredient codes (UNII)
- MLU3LU3EVV
- Status
- Authorised
- Status date
- 18 May 2020
- Authorisation date
- 18 May 2020
- Approved indications
- Zolgensma is indicated for the treatment of: patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1, or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.
- Product number
- EMEA/H/C/004750
- Authorisation holder
- Novartis Europharm Limited
- Priority medicines scheme
- Yes
- Generic
- No
- Biosimilar
- No
- Generic medicines
- 0
- Biosimilar medicines
- 0
- Advanced therapy
- Yes
- Conditional approval
- Yes
- Additional monitoring
- Yes
- Accelerated assessment
- No
- Exceptional circumstances
- No
Therapeutic areas
- Muscular Atrophy, Spinal
European medicines record
- Patents
- 0
- Designations
- 4
- Earliest recorded loss of exclusivity
- 24 Nov 2031
- First in class
- Yes
- Fast track
- No
- Breakthrough designation
- Yes
- Orphan designation
- Yes
- Priority review
- No
- Orphan exclusivity
- No
- Accelerated approval
- No
Mechanisms of action
- Mechanism
- Survival motor neuron protein exogenous gene
- Action type
- EXOGENOUS_GENE
- Target
- Survival motor neuron protein
- Protein identifier
- Q16637
Open Targets mechanisms
- Mechanism
- Survival motor neuron protein exogenous gene
- Action type
- EXOGENOUS GENE
- Target
- Survival motor neuron protein
- Condition
- Muscular Atrophy, Spinal
- Highest recorded phase
- 4
- Condition identifier
- EFO:0008525
- Subject identifier
- D009134
- Condition
- Spinal Muscular Atrophies of Childhood
- Highest recorded phase
- 3
- Condition identifier
- MONDO:0009669
- Subject identifier
- D014897
- Condition
- Spinal Muscular Atrophies of Childhood
- Highest recorded phase
- 3
- Condition identifier
- MONDO:0009673
- Subject identifier
- D014897
- Condition
- Muscular Atrophy, Spinal
- Highest recorded phase
- 4
- Condition identifier
- MONDO:0019079
- Subject identifier
- D009134
- Condition
- Muscular Atrophy, Spinal
- Highest recorded phase
- 4
- Condition identifier
- Orphanet:83419
- Subject identifier
- D009134
- Condition identifier
- EFO_0008525
- Condition identifier
- MONDO_0001516
- Highest recorded phase
- 4
- Condition identifier
- MONDO_0007027
- Highest recorded phase
- 1
- Condition identifier
- MONDO_0009669
- Highest recorded phase
- 1
- Condition identifier
- MONDO_0009669
- Highest recorded phase
- 3
- Condition identifier
- MONDO_0013209
- Highest recorded phase
- 1
- Condition identifier
- MONDO_0019079
- Highest recorded phase
- 3
- Event
- alanine aminotransferase increased
- Reports
- 74
- Reporting signal score
- 192.247
- Event
- aspartate aminotransferase increased
- Reports
- 80
- Reporting signal score
- 223.809
- Event
- atelectasis
- Reports
- 5
- Reporting signal score
- 9.80158
- Event
- blood creatine phosphokinase abnormal
- Reports
- 2
- Reporting signal score
- 7.62345
- Event
- blood creatine phosphokinase mb increased
- Reports
- 5
- Reporting signal score
- 17.4721
- Event
- blood creatinine decreased
- Reports
- 5
- Reporting signal score
- 15.3174
- Event
- blood lactate dehydrogenase increased
- Reports
- 7
- Reporting signal score
- 11.6696
- Event
- decreased appetite
- Reports
- 19
- Reporting signal score
- 14.6114
- Event
- decreased immune responsiveness
- Reports
- 3
- Reporting signal score
- 7.07941
- Event
- developmental hip dysplasia
- Reports
- 2
- Reporting signal score
- 6.50845
- Event
- diaphragmatic abnormal relaxation
- Reports
- 1
- Reporting signal score
- 6.84719
- Event
- enterovirus infection
- Reports
- 2
- Reporting signal score
- 6.97732
- Event
- gamma-glutamyltransferase increased
- Reports
- 8
- Reporting signal score
- 10.4956
- Event
- headache
- Reports
- 1
- Reporting signal score
- 8.87041
- Event
- hepatic enzyme increased
- Reports
- 50
- Reporting signal score
- 123.459
- Event
- increased bronchial secretion
- Reports
- 4
- Reporting signal score
- 14.3349
- Event
- liver function test increased
- Reports
- 38
- Reporting signal score
- 119.741
- Event
- mean cell haemoglobin concentration increased
- Reports
- 2
- Reporting signal score
- 7.73019
- Event
- monocyte count increased
- Reports
- 4
- Reporting signal score
- 10.4975
- Event
- myocardial hypoxia
- Reports
- 1
- Reporting signal score
- 7.35786
- Event
- oxygen saturation decreased
- Reports
- 8
- Reporting signal score
- 8.06259
- Event
- platelet count decreased
- Reports
- 42
- Reporting signal score
- 73.9638
- Event
- pneumonia mycoplasmal
- Reports
- 2
- Reporting signal score
- 6.72882
- Event
- pyrexia
- Reports
- 91
- Reporting signal score
- 138.664
- Event
- respiratory distress
- Reports
- 7
- Reporting signal score
- 9.50754
And 14 more, listed on the interactive page.
- NCT02122952
- NCT03306277
- NCT03461289
- NCT05335876
- NCT03381729
- NCT03505099
- NCT04042025
- Dose
- 1.1 × 10^14 vector genomes per kg
- Route
- Intravenous
- How often
- Single dose
- Discard if not infused within 8 hours of drawing into syringe
- Do not infuse as an IV push or bolus
- Infuse over 60 minutes
- Prodrug
- No
- Chirality
- Single enantiomer
- Inorganic
- No
- Natural product
- No
Sequences
- Component
- Sequence
- Type
- Nucleic Acid
- Sequence
- gcccaatacgcaaaccgcctctccccgcgcgttggccgattcattaatgcagctggcgtaatagcgaagaggcccgcaccgatcgcccttcccaacagttgcgcagcctgaatggcgaatggcgattccgttgcaatggctggcggtaatattgttctggatattaccagcaaggccgatagtttgagttcttctactcaggcaagtgatgttattactaatcaaagaagtattgcgacaacggttaatttgcgtgatggacagactcttttactcggtggcctcactgattataaaaacacttctcaggattctggcgtaccgttcctgtctaaaatccctttaatcggcctcctgtttagctcccgctctgattctaacgaggaaagcacgttatacgtgctcgtcaaagcaaccatagtacgcgccctgtagcggcgcattaagcgcggcgggtgtggtggttacgcgcagcgtgaccgctacacttgccagcgccctagcgcccgctcctttcgctttcttcccttcctttctcgccacgttcgccggctttccccgtcaagctctaaatcgggggctccctttagggttccgatttagtgctttacggcacctcgaccccaaaaaacttgattagggtgatggttcacgtagtgggccatcgccctgatagacggtttttcgccctttgacgttggagtccacgttctttaatagtggactcttgttccaaactggaacaacactcaaccctatctcggtctattcttttgatttataagggattttgccgatttcggcctattggttaaaaaatgagctgatttaacaaaaatttaacgcgaattttaacaaaatattaacgcttacaatttaaatatttgcttatacaatcttcctgtttttggggcttttctgattatcaaccggggtacatatgattgacatgctagttttacgattaccgttcatcgccctgcgcgctcgctcgctcactgaggccgcccgggcaaagcccgggcgtcgggcgacctttggtcgcccggcctcagtgagcgagcgagcgcgcagagagggagtggaattcacgcgtggatctgaattcaattcacgcgtggtacctctggtcgttacataacttacggtaaatggcccgcctggctgaccgcccaacgacccccgcccattgacgtcaataatgacgtatgttcccatagtaacgccaatagggactttccattgacgtcaatgggtggagtatttacggtaaactgcccacttggcagtacatcaagtgtatcatatgccaagtacgccccctattgacgtcaatgacggtaaatggcccgcctggcattatgcccagtacatgaccttatgggactttcctacttggcagtacatctactcgaggccacgttctgcttcactctccccatctcccccccctccccacccccaattttgtatttatttattttttaattattttgtgcagcgatgggggcggggggggggggggggcgcgcgccaggcggggcggggcggggcgaggggcggggcggggcgaggcggagaggtgcggcggcagccaatcagagcggcgcgctccgaaagtttccttttatggcgaggcggcggcggcggcggccctataaaaagcgaagcgcgcggcgggcgggagcgggatcagccaccgcggtggcggcctagagtcgacgaggaactgaaaaaccagaaagttaactggtaagtttagtctttttgtcttttatttcaggtcccggatccggtggtggtgcaaatcaaagaactgctcctcagtggatgttgcctttacttctaggcctgtacggaagtgttacttctgctctaaaagctgcggaattgtacccgcggccgatccaccggtccggaattcccgggatatcgtcgacccacgcgtccgggccccacgctgcgcacccgcgggtttgctatggcgatgagcagcggcggcagtggtggcggcgtcccggagcaggaggattccgtgctgttccggcgcggcacaggccagagcgatgattctgacatttgggatgatacagcactgataaaagcatatgataaagctgtggcttcatttaagcatgctctaaagaatggtgacatttgtgaaacttcgggtaaaccaaaaaccacacctaaaagaaaacctgctaagaagaataaaagccaaaagaagaatactgcagcttccttacaacagtggaaagttggggacaaatgttctgccatttggtcagaagacggttgcatttacccagctaccattgcttcaattgattttaagagagaaacctgtgttgtggtttacactggatatggaaatagagaggagcaaaatctgtccgatctactttccccaatctgtgaagtagctaataatatagaacagaatgctcaagagaatgaaaatgaaagccaagtttcaacagatgaaagtgagaactccaggtctcctggaaataaatcagataacatcaagcccaaatctgctccatggaactcttttctccctccaccaccccccatgccagggccaagactgggaccaggaaagccaggtctaaaattcaatggcccaccaccgccaccgccaccaccaccaccccacttactatcatgctggctgcctccatttccttctggaccaccaataattcccccaccacctcccatatgtccagattctcttgatgatgctgatgctttgggaagtatgttaatttcatggtacatgagtggctatcatactggctattatatgggttttagacaaaatcaaaaagaaggaaggtgctcacattccttaaattaaggagaaatgctggcatagagcagcactaaatgacaccactaaagaaacgatcagacagatctagaaagcttatcgataccgtcgactagagctcgctgatcagcctcgactgtgccttctagttgccagccatctgttgtttgcccctcccccgtgccttccttgaccctggaaggtgccactcccactgtcctttcctaataaaatgaggaaattgcatcgcattgtctgagtaggtgtcattctattctggggggtggggtggggcaggacagcaagggggaggattgggaagacaatagcaggcatgctggggagagatcgatctgaggaacccctagtgatggagttggccactccctctctgcgcgctcgctcgctcactgaggccgggcgaccaaaggtcgcccgacgcccgggctttgcccgggcggcctcagtgagcgagcgagcgcgcagagagggagtggcccccccccccccccccccggcgattctcttgtttgctccagactctcaggcaatgacctgatagcctttgtagagacctctcaaaaatagctaccctctccggcatgaatttatcagctagaacggttgaatatcatattgatggtgatttgactgtctccggcctttctcacccgtttgaatctttacctacacattactcaggcattgcatttaaaatatatgagggttctaaaaatttttatccttgcgttgaaataaaggcttctcccgcaaaagtattacagggtcataatgtttttggtacaaccgatttagctttatgctctgaggctttattgcttaattttgctaattctttgccttgcctgtatgatttattggatgttggaatcgcctgatgcggtattttctccttacgcatctgtgcggtatttcacaccgcatatggtgcactctcagtacaatctgctctgatgccgcatagttaagccagccccgacacccgccaacactatggtgcactctcagtacaatctgctctgatgccgcatagttaagccagccccgacacccgccaacacccgctgacgcgccctgacgggcttgtctgctcccggcatccgcttacagacaagctgtgaccgtctccgggagctgcatgtgtcagaggttttcaccgtcatcaccgaaacgcgcgagacgaaagggcctcgtgatacgcctatttttataggttaatgtcatgataataatggtttcttagacgtcaggtggcacttttcggggaaatgtgcgcggaacccctatttgtttatttttctaaatacattcaaatatgtatccgctcatgagacaataaccctgataaatgcttcaataatattgaaaaaggaagagtatgagtattcaacatttccgtgtcgcccttattcccttttttgcggcattttgccttcctgtttttgctcacccagaaacgctggtgaaagtaaaagatgctgaagatcagttgggtgcacgagtgggttacatcgaactggatctcaacagcggtaagatccttgagagttttcgccccgaagaacgttttccaatgatgagcacttttaaagttctgctatgtggcgcggtattatcccgtattgacgccgggcaagagcaactcggtcgccgcatacactattctcagaatgacttggttgagtactcaccagtcacagaaaagcatcttacggatggcatgacagtaagagaattatgcagtgctgccataaccatgagtgataacactgcggccaacttacttctgacaacgatcggaggaccgaaggagctaaccgcttttttgcacaacatgggggatcatgtaactcgccttgatcgttgggaaccggagctgaatgaagccataccaaacgacgagcgtgacaccacgatgcctgtagcaatggcaacaacgttgcgcaaactattaactggcgaactacttactctagcttccggcaacaattaatagactggatggaggcggataaagttgcaggaccacttctgcgctcggcccttccggctggctggtttattgctgataaatctggagccggtgagcgtgggtctcgcggtatcattgcagcactggggccagatggtaagccctcccgtatcgtagttatctacacgacggggagtcaggcaactatggatgaacgaaatagacagatcgctgagataggtgcctcactgattaagcattggtaactgtcagaccaagtttactcatatatactttagattgatttaaaacttcatttttaatttaaaaggatctaggtgaagatcctttttgataatctcatgaccaaaatcccttaacgtgagttttcgttccactgagcgtcagaccccgtagaaaagatcaaaggatcttcttgagatcctttttttctgcgcgtaatctgctgcttgcaaacaaaaaaaccaccgctaccagcggtggtttgtttgccggatcaagagctaccaactctttttccgaaggtaactggcttcagcagagcgcagataccaaatactgttcttctagtgtagccgtagttaggccaccacttcaagaactctgtagcaccgcctacatacctcgctctgctaatcctgttaccagtggctgctgccagtggcgataagtcgtgtcttaccgggttggactcaagacgatagttaccggataaggcgcagcggtcgggctgaacggggggttcgtgcacacagcccagcttggagcgaacgacctacaccgaactgagatacctacagcgtgagctatgagaaagcgccacgcttcccgaagggagaaaggcggacaggtatccggtaagcggcagggtcggaacaggagagcgcacgagggagcttccagggggaaacgcctggtatctttatagtcctgtcgggtttcgccacctctgacttgagcgtcgatttttgtgatgctcgtcaggggggcggagcctatggaaaaacgccagcaacgcggcctttttacggttcctggccttttgctggccttttgctcacatgttctttcctgtttatcccctgattctgtggataaccgtattaccgcctttgagtgagctgataccgctcgccgcagccgaacgaccgagcgcagcgagtcagtgagcgaggaagcggaagagc
- 71894-200
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Approved for (3)
- Proximal spinal muscular atrophy
- Spinal muscular atrophy
- Spinal muscular atrophy, type III
In development for (2)
- Spinal muscular atrophy, type 1 Phase 3
- Spinal muscular atrophy, type II Phase 3