| Status: Excluded due to NICE appraisal | |
Product meets AWMSG exclusion criteria due to NICE appraisal HST15: Onasemnogene abeparvovec for treating spinal muscular atrophy and HST24: Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophy |
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Medicine details |
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| Medicine name | onasemnogene abeparvovec (Zolgensma®) |
| Formulation | solution for infusion |
| Reference number | 3831 |
| Indication | 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 |
| Company | AveXis |
| BNF chapter | Musculoskeletal & joint diseases |
| Submission type | N/A |
| Status | Excluded due to NICE appraisal |
| Date of issue | 19/04/2023 |
| NICE guidance | HST15: Onasemnogene abeparvovec for treating spinal muscular atrophy HST24: Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophy |