Zolgensma which has a reported list price of £1.79 million( ₹18 crore) per dose is labelled the most expensive drug in the world. Zolgensma was approved by the NHS England on Monday to treat babies with a rare fatal genetic disorder.
Zolgensma, a life-saving drug, can enable mobility in babies and young children suffering from a rare genetic condition. Zolgensma is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patient less than 2 years of age with spinal muscular atrophy (SMA).
The one-off gene therapy treats SMA, a rare and often fatal genetic disease that causes paralysis, muscle weakness and progressive loss of movement.
Babies born with severe type 1 SMA – the most common form of the condition – have a life expectancy of just two years.
In studies, Zolgensma, manufactured by Novartis Gene Therapies, has helped babies to reach milestones such as breathe without a ventilator, sit up on their own and crawl and walk after a single infusion treatment.
The latest data suggested that Zolgensma can provide rapid and sustained improvement in motor function for young children with type 1 SMA and prolong their lives.
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