Great Britain
Paid by the state: Baby receives the most expensive drug in the world
Five-month-old Arthur Morgan received the rescue Zolgensma infusion at the end of May. (Symbol image)
© SURAKIT SAWANGCHIT / Shutterstock
The drug Zolgensma is the most expensive drug in the world – and so far the only one that can help with spinal muscular atrophy. Now the UK’s national health care system, the NHS, has paid for the treatment.
Little Arthur Morgan from south London is only five months old and seriously ill: he suffers from spinal muscular atrophy (SMA), a genetic defect also known as muscle wasting. Those affected usually suffer from progressive muscle weakness, loss of movement and breathing problems. In the worst case, the disease can be fatal: More than half of the babies affected die before they are two years old.
Arthur’s parents had noticed that something was wrong with their young son when, unlike other children his age, he was unable to sit up straight and had difficulty holding his head. In the hospital, the family was then diagnosed with SMA. But there was hope, because there is a saving drug: Zolgensma should be able to prevent paralysis due to SMA – but at 1.79 million pounds (over two million euros) it is the most expensive drug in the world and has been for many in the past Families unaffordable.
Health care system NHS provides Zolgensma
Little Arthur was lucky, however: barely three weeks after the diagnosis, he received the life-saving infusion of Zolgensma – because the British National Health Service (NHS) had already signed a contract with the manufacturer Novatis Gene Therapies in March and was able to do so Offer Arthur the medicine he needs. The purchase price agreed between the NHS and Novatis has not been published, but it is said that the amount is “fair” to British taxpayers. For Arthur’s family, the NHS drug delivery means hope for their son: “When we found out Arthur was going to get the treatment and would be the first patient, I just collapsed,” Father Reece told the Press Association. “It has been a turbulent couple of weeks, filled with many fears and changes, since we found out about his illness and what it could mean for him and our family.” He was “so grateful” to the NHS.
A single infusion is sufficient
Treatment with Zolgensma is a one-time treatment and is a gene therapy. A faulty gene that patients carry and that prevents the body from producing a protein that is important for muscle function is replaced by a corrected gene via infusion. This then instructs the body to produce the protein it needs. According to studies, a single treatment with Zolgensma in infants ensures that they can learn to sit, crawl and walk and no longer need a ventilator.
Long-term consequences of gene therapy are not yet known – it was only approved for a short time. Little Arthur, who was born six weeks early, has SMA type 1, the most common form of the disease. SMA is a genetic disease and is inherited.
Arthur’s family has now opened a donation page. Already 25,000 pounds (almost 30,000 euros) have been collected in this way so far. The money is intended to help Arthur’s parents take care of both their sick son and older sister Nancy without having to worry about money during times of intensive hospitalization. “We still don’t know what the future will bring, but this treatment gives Arthur the best possible chance for the best possible future,” Father Reece tells the Press Association, adding, “This is the best possible treatment and will give him the best possible life give what he can have and we are so grateful that the NHS is there for him. ” The British state health system will assume the costs of other affected babies such as Arthur in Great Britain in the future.
Correction: A previous version of the article stated that Arthur was the first patient to receive a Zolgensma infusion. That’s not true.
swell: “Press Assocation” in “The Guardian”
This article originally appeared on stern.de.