The United Kingdom's National Health Service (NHS) has approved the 'most expensive drug in the world' to cure a rare genetic disorder. The one-off gene therapy Zolgensma, manufactured by Novartis Gene Therapies, has a reported list price of ₹18 crore (£1.79 million) per dose, the health service said in a statement Monday.
The medicine treats Spinal Muscular Atrophy (SMA), a rare and often fatal genetic disease that causes paralysis, muscle weakness and progressive loss of movement. It will be used for babies and young children suffering from SMA. Babies born with severe type 1 SMA – the most common form of the condition – have a life expectancy of just two years.
“This deal is a life-changer for youngsters with this cruel disease and for their families," NHS England chief executive Sir Simon Stevens said.
“Spinal Muscular Atrophy is the leading genetic cause of death among babies and young children, which is why NHS England has moved mountains to make this treatment available, while successfully negotiating hard behind the scenes to ensure a price that is fair to taxpayers," CEO added.