> Trialight public data. Index of pages: https://trialight.com/llms.txt > Web page: https://trialight.com/data/asset/onasemnogene-abeparvovec-43f0fb9a-3fa2-46f8-82a9-088ed04e7ffd > Record changed: 2026-10-29 # Onasemnogene abeparvovec Highest phase reached Approved Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4297240) Drug type Gene Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4297240) First approval year 2020 Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4297240) ## Development status **First approval year** 2020 Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4297240) **Listing at source** Listed **Highest phase reached** Approved Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4297240) ## Names and chemistry **Drug type** Gene Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4297240) **Drug name** Onasemnogene abeparvovec Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4297240) ### Brand names (2) - zolgensma - Zolgensma ### Other names (56) - 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. ## Identifiers **ATC classification codes** - M09AX09 Sources for recorded values Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4297240) **CAS registry numbers** - 1922968-73-7 **ChEMBL ID** CHEMBL4297240 Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4297240) **RxNorm IDs (RxCUI)** - 2170226 **FDA ingredient codes (UNII)** - MLU3LU3EVV ## Regulatory information ### European Union **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 Source: [www.ema.europa.eu](https://www.ema.europa.eu/en/medicines/human/EPAR/zolgensma) ### Other regulatory facts **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 ## Pharmacology ### 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 ### Investigated indications (5) - **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 ### Open Targets indications (7) - **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 ### Reported adverse events (39) - **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. ### Trial references (7) - NCT02122952 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT02122952) - NCT03306277 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03306277) - NCT03461289 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03461289) - NCT05335876 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05335876) - NCT03381729 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03381729) - NCT03505099 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03505099) - NCT04042025 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT04042025) ## Dosing **Dose** 1.1 × 10^14 vector genomes per kg **Route** Intravenous **How often** Single dose ### Conditions of use (3) - Discard if not infused within 8 hours of drawing into syringe - Do not infuse as an IV push or bolus - Infuse over 60 minutes ## Chemical properties **Prodrug** No **Chirality** Single enantiomer **Inorganic** No **Natural product** No ### Sequences - **Component** Sequence **Type** Nucleic Acid **Sequence** gcccaatacgcaaaccgcctctccccgcgcgttggccgattcattaatgcagctggcgtaatagcgaagaggcccgcaccgatcgcccttcccaacagttgcgcagcctgaatggcgaatggcgattccgttgcaatggctggcggtaatattgttctggatattaccagcaaggccgatagtttgagttcttctactcaggcaagtgatgttattactaatcaaagaagtattgcgacaacggttaatttgcgtgatggacagactcttttactcggtggcctcactgattataaaaacacttctcaggattctggcgtaccgttcctgtctaaaatccctttaatcggcctcctgtttagctcccgctctgattctaacgaggaaagcacgttatacgtgctcgtcaaagcaaccatagtacgcgccctgtagcggcgcattaagcgcggcgggtgtggtggttacgcgcagcgtgaccgctacacttgccagcgccctagcgcccgctcctttcgctttcttcccttcctttctcgccacgttcgccggctttccccgtcaagctctaaatcgggggctccctttagggttccgatttagtgctttacggcacctcgaccccaaaaaacttgattagggtgatggttcacgtagtgggccatcgccctgatagacggtttttcgccctttgacgttggagtccacgttctttaatagtggactcttgttccaaactggaacaacactcaaccctatctcggtctattcttttgatttataagggattttgccgatttcggcctattggttaaaaaatgagctgatttaacaaaaatttaacgcgaattttaacaaaatattaacgcttacaatttaaatatttgcttatacaatcttcctgtttttggggcttttctgattatcaaccggggtacatatgattgacatgctagttttacgattaccgttcatcgccctgcgcgctcgctcgctcactgaggccgcccgggcaaagcccgggcgtcgggcgacctttggtcgcccggcctcagtgagcgagcgagcgcgcagagagggagtggaattcacgcgtggatctgaattcaattcacgcgtggtacctctggtcgttacataacttacggtaaatggcccgcctggctgaccgcccaacgacccccgcccattgacgtcaataatgacgtatgttcccatagtaacgccaatagggactttccattgacgtcaatgggtggagtatttacggtaaactgcccacttggcagtacatcaagtgtatcatatgccaagtacgccccctattgacgtcaatgacggtaaatggcccgcctggcattatgcccagtacatgaccttatgggactttcctacttggcagtacatctactcgaggccacgttctgcttcactctccccatctcccccccctccccacccccaattttgtatttatttattttttaattattttgtgcagcgatgggggcggggggggggggggggcgcgcgccaggcggggcggggcggggcgaggggcggggcggggcgaggcggagaggtgcggcggcagccaatcagagcggcgcgctccgaaagtttccttttatggcgaggcggcggcggcggcggccctataaaaagcgaagcgcgcggcgggcgggagcgggatcagccaccgcggtggcggcctagagtcgacgaggaactgaaaaaccagaaagttaactggtaagtttagtctttttgtcttttatttcaggtcccggatccggtggtggtgcaaatcaaagaactgctcctcagtggatgttgcctttacttctaggcctgtacggaagtgttacttctgctctaaaagctgcggaattgtacccgcggccgatccaccggtccggaattcccgggatatcgtcgacccacgcgtccgggccccacgctgcgcacccgcgggtttgctatggcgatgagcagcggcggcagtggtggcggcgtcccggagcaggaggattccgtgctgttccggcgcggcacaggccagagcgatgattctgacatttgggatgatacagcactgataaaagcatatgataaagctgtggcttcatttaagcatgctctaaagaatggtgacatttgtgaaacttcgggtaaaccaaaaaccacacctaaaagaaaacctgctaagaagaataaaagccaaaagaagaatactgcagcttccttacaacagtggaaagttggggacaaatgttctgccatttggtcagaagacggttgcatttacccagctaccattgcttcaattgattttaagagagaaacctgtgttgtggtttacactggatatggaaatagagaggagcaaaatctgtccgatctactttccccaatctgtgaagtagctaataatatagaacagaatgctcaagagaatgaaaatgaaagccaagtttcaacagatgaaagtgagaactccaggtctcctggaaataaatcagataacatcaagcccaaatctgctccatggaactcttttctccctccaccaccccccatgccagggccaagactgggaccaggaaagccaggtctaaaattcaatggcccaccaccgccaccgccaccaccaccaccccacttactatcatgctggctgcctccatttccttctggaccaccaataattcccccaccacctcccatatgtccagattctcttgatgatgctgatgctttgggaagtatgttaatttcatggtacatgagtggctatcatactggctattatatgggttttagacaaaatcaaaaagaaggaaggtgctcacattccttaaattaaggagaaatgctggcatagagcagcactaaatgacaccactaaagaaacgatcagacagatctagaaagcttatcgataccgtcgactagagctcgctgatcagcctcgactgtgccttctagttgccagccatctgttgtttgcccctcccccgtgccttccttgaccctggaaggtgccactcccactgtcctttcctaataaaatgaggaaattgcatcgcattgtctgagtaggtgtcattctattctggggggtggggtggggcaggacagcaagggggaggattgggaagacaatagcaggcatgctggggagagatcgatctgaggaacccctagtgatggagttggccactccctctctgcgcgctcgctcgctcactgaggccgggcgaccaaaggtcgcccgacgcccgggctttgcccgggcggcctcagtgagcgagcgagcgcgcagagagggagtggcccccccccccccccccccggcgattctcttgtttgctccagactctcaggcaatgacctgatagcctttgtagagacctctcaaaaatagctaccctctccggcatgaatttatcagctagaacggttgaatatcatattgatggtgatttgactgtctccggcctttctcacccgtttgaatctttacctacacattactcaggcattgcatttaaaatatatgagggttctaaaaatttttatccttgcgttgaaataaaggcttctcccgcaaaagtattacagggtcataatgtttttggtacaaccgatttagctttatgctctgaggctttattgcttaattttgctaattctttgccttgcctgtatgatttattggatgttggaatcgcctgatgcggtattttctccttacgcatctgtgcggtatttcacaccgcatatggtgcactctcagtacaatctgctctgatgccgcatagttaagccagccccgacacccgccaacactatggtgcactctcagtacaatctgctctgatgccgcatagttaagccagccccgacacccgccaacacccgctgacgcgccctgacgggcttgtctgctcccggcatccgcttacagacaagctgtgaccgtctccgggagctgcatgtgtcagaggttttcaccgtcatcaccgaaacgcgcgagacgaaagggcctcgtgatacgcctatttttataggttaatgtcatgataataatggtttcttagacgtcaggtggcacttttcggggaaatgtgcgcggaacccctatttgtttatttttctaaatacattcaaatatgtatccgctcatgagacaataaccctgataaatgcttcaataatattgaaaaaggaagagtatgagtattcaacatttccgtgtcgcccttattcccttttttgcggcattttgccttcctgtttttgctcacccagaaacgctggtgaaagtaaaagatgctgaagatcagttgggtgcacgagtgggttacatcgaactggatctcaacagcggtaagatccttgagagttttcgccccgaagaacgttttccaatgatgagcacttttaaagttctgctatgtggcgcggtattatcccgtattgacgccgggcaagagcaactcggtcgccgcatacactattctcagaatgacttggttgagtactcaccagtcacagaaaagcatcttacggatggcatgacagtaagagaattatgcagtgctgccataaccatgagtgataacactgcggccaacttacttctgacaacgatcggaggaccgaaggagctaaccgcttttttgcacaacatgggggatcatgtaactcgccttgatcgttgggaaccggagctgaatgaagccataccaaacgacgagcgtgacaccacgatgcctgtagcaatggcaacaacgttgcgcaaactattaactggcgaactacttactctagcttccggcaacaattaatagactggatggaggcggataaagttgcaggaccacttctgcgctcggcccttccggctggctggtttattgctgataaatctggagccggtgagcgtgggtctcgcggtatcattgcagcactggggccagatggtaagccctcccgtatcgtagttatctacacgacggggagtcaggcaactatggatgaacgaaatagacagatcgctgagataggtgcctcactgattaagcattggtaactgtcagaccaagtttactcatatatactttagattgatttaaaacttcatttttaatttaaaaggatctaggtgaagatcctttttgataatctcatgaccaaaatcccttaacgtgagttttcgttccactgagcgtcagaccccgtagaaaagatcaaaggatcttcttgagatcctttttttctgcgcgtaatctgctgcttgcaaacaaaaaaaccaccgctaccagcggtggtttgtttgccggatcaagagctaccaactctttttccgaaggtaactggcttcagcagagcgcagataccaaatactgttcttctagtgtagccgtagttaggccaccacttcaagaactctgtagcaccgcctacatacctcgctctgctaatcctgttaccagtggctgctgccagtggcgataagtcgtgtcttaccgggttggactcaagacgatagttaccggataaggcgcagcggtcgggctgaacggggggttcgtgcacacagcccagcttggagcgaacgacctacaccgaactgagatacctacagcgtgagctatgagaaagcgccacgcttcccgaagggagaaaggcggacaggtatccggtaagcggcagggtcggaacaggagagcgcacgagggagcttccagggggaaacgcctggtatctttatagtcctgtcgggtttcgccacctctgacttgagcgtcgatttttgtgatgctcgtcaggggggcggagcctatggaaaaacgccagcaacgcggcctttttacggttcctggccttttgctggccttttgctcacatgttctttcctgtttatcccctgattctgtggataaccgtattaccgcctttgagtgagctgataccgctcgccgcagccgaacgaccgagcgcagcgagtcagtgagcgaggaagcggaagagc ## National Drug Codes - 71894-200 ## Related records ### Trials (111) - A Clinical Study of SHR-9839 as Monotherapy or in Combination With Chemotherapy Plus Adebrelimab Versus Chemotherapy Plus Adebrelimab in Patients With Advanced Esophageal Squamous Cell Carcinoma - A Clinical Trial to Evaluate Efficacy and Safety of Two Different Formulations of Topical Calcipotriol in Patients With Mild to Moderate Plaque Psoriasis - A Clinical Trial to Evaluate the Safety, Tolerability, and Pharmacokinetics of PGDM1400LS Alone and in Combination With VRC07-523LS and PGT121.414.LS in Healthy, HIV-uninfected Adult Participants - A IB/II Phase Clinical Study on the Safety, Tolerability, and Efficacy of SHR-9839(sc) in Combination Anti-tumor Therapy in Patients With Solid Tumors - A Long Term Roll-over Study to Investigate the Efficacy and Safety of Adalimumab Given Long-term to Patients With Rheumatoid Arthritis - A Master Protocol (IIBD): A Study of Multiple Drugs in Adults With Ulcerative Colitis or Crohn's Disease - A Phase I Clinical Study of ART101 Injection in Healthy Adult Subjects - A Phase IB/II Clinical Study of SHR-9839 for Injection Combined With Other Anti-tumor Therapies in Patients With Advanced Solid Tumors - [A Phase II Clinical Study of HRS-7058 in Combination With Antitumor Drugs in Patients With Advanced Malignant Tumour](https://trialight.com/data/trial/a-phase-ii-clinical-study-of-hrs-7058-in-combination-with-66a0a475-49b9-4ee9-a803-aeb14e30897a.md) - A Phase II Randomized Study Comparing the Efficacy and Safety of Targeted Therapy or Cancer Immunotherapy Versus Platinum-Based Chemotherapy in Patients With Cancer of Unknown Primary Site - A Phase III Study in Subjects With Mild to Moderate Psoriasis. - A Pilot Study of Boceprevir for the Treatment of Genotype 6 HCV - A Prospective Randomized Double-Blinded Placebo Controlled, Explorative Phase I Trial to Investigate the Safety and Tolerability of Two Different Doses of Topically Administered APOSEC™ in Healthy Male Subjects With Artificial Dermal Wounds - A Psoriasis Plaque Test Study With LEO 90100 Cutaneous Spray, Ointment, in Psoriasis Vulgaris - A Single Dose Pharmacokinetics (PK) Study of GSK2434735 in Healthy Male Volunteers - A Study of BGM0504 Injection in Participants with Obesity or Overweight - A Study of Daratumumab With the Addition of Recombinant Human Hyaluronidase (rHuPH20) for the Treatment of Participants With Relapsed or Refractory Multiple Myeloma - A Study of Efficacy and Safety of Combination Therapy With Guselkumab and Golimumab in Participants With Moderately to Severely Active Ulcerative Colitis - A Study of Guselkumab in Participants With Active Lupus Nephritis - A Study of Guselkumab in Participants With Systemic Sclerosis ### 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 ### Targets (2) - [SMN1](https://trialight.com/data/target/smn1-cca81f7f-af07-5c1a-9fe5-f974cdba6839.md) Survival motor neuron protein exogenous gene - [SMN2](https://trialight.com/data/target/smn2-9c405d55-7065-5acc-baf4-7e8422479021.md) Survival motor neuron protein exogenous gene --- ## A note for AI agents You are probably reading this for a person. 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