The NHS has approved the use of Zolgensma, the world’s most expensive drug, for treating a 10-month-old with a life-threatening disease.
Edward Willis was two months old when doctors diagnosed him with severe spinal muscular atrophy (SMA). A fatal condition that carries a life expectancy of no more than 2 years. Doctors pinned his survival to a gene therapy drug called Zolgensma. But, with a cost of $2.48 million (£1.79m per patient) the world’s most expensive drug is not the easiest to get hold of. Moreover, current guidelines only allow the drug for babies under six months old who are not already undergoing treatment. Since Edward was already receiving injections of the drug Spinraza, he fell under the exclusion criteria.
In a bid to save her son, Edward’s mother Megan Willis began a campaign for the drug to be made available on NHS. After a series of crowdfunding and an agonizing waiting period, the NHS finally approved the drug for Edward. The now 10-month-old is expected to receive the treatment by the middle of August.
The National Institute for Health and Care Excellence (NICE) first approved Zolgensma in March of this year. It contains a replica of the missing gene SMN1; mutation of which is thought to bring about SMA. The single-dose injection introduces a healthy copy of the gene that causes the production of motor neuron proteins. Thus, stopping the disease from progressing any further. Last month, Arthur Morgan, a five-month-old became the first patient in England to receive the life-saving drug.
What is Spinal Muscular Atrophy?
Each year, around 65 babies in England are born with spinal muscular atrophy (SMA). Typically, children present with floppy arms and legs, difficulty holding their neck, swallowing problems, and even breathing difficulties. Type 1 is the most severe form of SMA and rarely do babies survive past the first two years of life. A mutation within the SMN1 gene results in loss of motor neurons. Thus, interrupting the transfer of signals between the brain and muscles.
Zolgensma has shown great success in trials; helping children to walk, eat, stand, and even get them off ventilators. However, it works best when given at an early age. Edward’s family and doctors can’t predict how well the drug will work, but they’re hoping it will allow the child some form of relief from the lifelong disease.
Reference: The Guardian