Onasemnogene abeparvovec is a gene therapy designed to introduce a functional copy of the survival motor neuron gene (SMN1) in the transduced cells to address the monogenic root cause of the disease. By providing an alternative source of SMN protein expression in motor neurons, it is expected to promote the survival and function of transduced motor neurons.
This medicinal substance has been classified in the anatomical therapeutic chemical (ATC) classification according to its main therapeutic use as follows:
| ATC code | Group title | Classification |
|---|---|---|
| M09AX09 | M Musculo-skeletal system → M09 Other drugs for disorders of the musculo-skeletal system → M09A Other drugs for disorders of the musculo-skeletal system → M09AX Other drugs for disorders of the musculo-skeletal system | |
Competent medicine agencies globally have authorized commercialization of this active ingredient according to these medication package inserts (MPIs):
| Title | Information Source | Document Type | |
|---|---|---|---|
| ZOLGENSMA Solution for infusion | European Medicines Agency (EU) | MPI, EU: SmPC |
Onasemnogene abeparvovec is an active ingredient of these brands:
Austria (AT)Brazil (BR)Croatia (HR)Estonia (EE)Finland (FI)France (FR)Ireland (IE)Israel (IL)Italy (IT)Lithuania (LT)Poland (PL)Romania (RO)United Kingdom (UK)Note the following: The list of brand names is continuously updated, and thus does not include the total of products circulating worldwide. |
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