Zolgensma Injection Prescription Required

Zolgensma Injection

Introduction

Zolgensma Injection contains the active ingredient Onasemnogene Abeparvovec-xioi and is developed by Novartis Gene Therapies (formerly AveXis). It is a one-time gene therapy and represents one of the most significant advances in the treatment of spinal muscular atrophy (SMA), a rare and devastating inherited neuromuscular disease. SMA is caused by mutations or deletion of the SMN1 (survival of motor neuron 1) gene, resulting in progressive loss of motor neurons and severe muscle weakness that can be fatal, particularly in infants with SMA type 1.

Zolgensma Injection works by using an adeno-associated virus serotype 9 (AAV9) vector to deliver a functional copy of the human SMN1 gene into the motor neurons of the central nervous system. Once the gene reaches the motor nerve cells, it begins to produce the survival motor neuron (SMN) protein, which is absent or severely deficient in SMA patients. By restoring SMN protein production in motor neurons, Zolgensma can halt or substantially slow the progression of motor neuron loss, enabling treated patients to achieve motor milestones that would otherwise be impossible.

Zolgensma is given as a single, one-time intravenous infusion. It is approved for paediatric patients with SMA with bi-allelic mutations in the SMN1 gene, and is most effective when administered early ideally before the onset of significant symptom progression, including in pre-symptomatic newborns identified through newborn screening.

Uses

Zolgensma Injection is used for:

• Treatment of spinal muscular atrophy (SMA) in paediatric patients with bi-allelic mutations in the SMN1 gene.

• One-time gene replacement therapy to restore survival motor neuron (SMN) protein production in motor neurons.

• Prevention of progressive motor neuron loss in SMA type 1 and other SMA subtypes in eligible paediatric patients.

Storage

• Store in an ultra-low temperature freezer at -90°C to -60°C at the treatment centre.

• Do not freeze and thaw repeatedly.

• The thawed product should be used within the timeframe specified in the prescribing information.

• Must be handled and administered exclusively by trained healthcare professionals in a clinical setting.

• Keep out of reach of children.

Disclaimer

MrMed’s primary intention is to ensure that its consumers get information that is reviewed by experts, accurate and trustworthy. The information and contents of this website are for informational purposes only. They are not intended to be a substitute for professional medical advice, diagnosis, or treatment. Please seek the advice of your doctor and discuss all your queries related to any disease or medicine. Do not disregard professional medical advice or delay in seeking it because of something you have read on MrMed. Our mission is to support, not replace, the doctor-patient relationship