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UK regulator approves world’s ‘most expensive drug’ priced at $2.48 million per dose to treat rare disorder

The United Kingdom’s drug regulator approved the world’s ‘most expensive drug’ which is used to treat a rare genetic disorder.

Manufactured by Novartis Gene Therapies, Zolgensma, has a list price of approximately USD $2.48 million per dose, the National Health Services England said.

The medicine is used to treat Spinal Muscular Atrophy (SMA), a rare and often fatal genetic disease that causes paralysis, muscle weakness and progressive loss of movement.




Zolgensma will be used on babies and young children suffering the genetic disorder. Babies born with severe type 1 SMA have a life expectancy of just two years.

Studies have shown encouraging results as Zolgensma helped babies to breathe without a ventilator, sit up on their own and crawl and walk after a single infusion treatment. The treatment contains a replica of the mussing gene SMN1 and is given as a single intravenous infusion, the NHS said.

The active ingredient onasemnogene abeparvovec goes into the nerves and restores the deformed gene, which then produces proteins essential for nerve function and controlling muscle movement.



“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.