ZOLGENSMA® (onasemnogene abeparvovec-xioi)

Indications and Usage:

ZOLGENSMA is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

Limitations of Use

  • The safety and effectiveness of repeat administration of ZOLGENSMA have not been evaluated.
  • The use of ZOLGENSMA in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator-dependence) has not been evaluated.
Prescribing Information