ZOLGENSMA (onasemnogene abeparvovec-xioi)

How It Works

Zolgensma works by delivering a functional copy of the SMN gene into the patient’s cells using a viral vector called adeno-associated virus 9 (AAV9). This vector targets motor neuron cells and introduces the healthy SMN gene, which then produces the survival motor neuron (SMN) protein. This protein is essential for the maintenance and function of motor neurons, and its presence helps to improve muscle function and slow the progression of SMA.

Side Effects

• Elevated liver enzymes
• Nausea and vomiting
• Decreased platelet count (thrombocytopenia)
• Fever
• Decreased appetite
• Elevated cardiac troponin-I

Indications

• Treatment of spinal muscular atrophy (SMA) in pediatric patients under the age of 2 with bi-allelic mutations in the SMN1 gene

Contraindications

• Hypersensitivity to any of the components of Zolgensma
• Patients with advanced liver disease
• Severe pre-existing thrombocytopenia

Price Comparison in Different Countries

Country	Price	Reference
Pakistan	Approximately PKR 250,000,000	Dawaai Pakistan
United States	USD 2,125,000	GoodRx
United Kingdom	GBP 1,795,000	NHS UK
Canada	CAD 2,850,000	Health Canada
Australia	AUD 2,900,000	PBS Australia

Top 5 Global Brands

1. Novartis
2. Pfizer
3. Roche
4. Sanofi
5. GlaxoSmithKline (GSK)

Conclusion

Zolgensma (Onasemnogene Abeparvovec-xioi) represents a significant advancement in the treatment of spinal muscular atrophy (SMA). By providing a functional copy of the SMN gene, Zolgensma helps improve motor neuron function and slow the progression of SMA in young children. Despite its high cost, Zolgensma offers hope for better quality of life for patients with SMA. The cost of Zolgensma varies widely across different countries, reflecting disparities in healthcare pricing and accessibility. Patients and families should consult with their healthcare providers and explore financial assistance programs to access this life-changing treatment.