Zolgensma™ (onasemnogene abeparvovec-xioi) is the second treatment approved for SMA in Europe and the first gene therapy for the condition.
The European Commission granted conditional approval for Zolgensma for the treatment of patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and:
The approval covers babies and young children with SMA up to 21 kg according to the approved dosing guidance.
Zolgensma™ is a gene therapy designed to address the genetic root cause of SMA by replacing the function of the missing or faulty SMN1 gene. Administered during a single, intravenous (IV) infusion, Zolgensma delivers a new working copy of the SMN1 gene into a patient’s cells, halting disease progression.
Zolgensma™ is made up of a new, working copy of a human SMN gene that is placed inside a vector. This vector transports the new, working SMN gene to the motor neuron cells in the body.
The vector that delivers the SMN gene is made from a virus called adeno-associated virus 9, or AAV9. This type of virus is harmless. To make the vector, the DNA of the virus is removed and the new SMN gene inserted in its place. Vectors are used because they can travel throughout the body and deliver the new, working gene to the cells where it is needed.
When the new gene reaches its destination, it is ready to tell the motor neurons to start making SMN protein. This happens throughout the body, with many vectors delivering a new, working copy of the SMN gene to motor neurons. Motor neurons now make sufficient SMN protein to survive, function, and be maintained.