The world’s most expensive drug, Zolgensma has a reported list price of nearly £1.8m per dose been licensed for use on the NHS in England, says an article published in Sky News.
Reported List price
Zolgensma has a reported list price of nearly £1.8m per dose, but the health service said it had struck a “landmark confidential deal” with its maker at a “price that is fair to taxpayers”.
SMA Treatment
The one-time gene therapy will be used to treat Spinal Muscular Atrophy (SMA) in infants. The often fatal muscle condition affects nerves in the spinal cord, leading to paralysis.
In the UK, up to 80 babies a year are born with SMA. Children who have the most common form of it – type 1 – have a life expectancy of only two years.
Zolgensma Working
Zolgensma, which is made by Novartis Gene Therapies, replicates a missing gene and restores nerve and muscle function.
Studies have shown it can help infants to breathe without ventilators, sit up unaided, crawl and walk.
The National Institute for Health and Care Excellence (NICE) said there was “evidence of exceptional benefit to young babies, potentially allowing them to reach normal childhood developmental milestones”.
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Source : Sky News
