Spinal muscular atrophy (SMA) is a debilitating condition that affects around one in every 10,000 children.
Zolgensma, the most effective treatment, is also the world’s most expensive drug.
A single one-time infusion of the drug costs Dh8 million.
But why does it cost so much?
The National explains.
What is SMA?
SMA is a hereditary disease caused by a missing or faulty gene that the body requires to make a protein essential for motor neuron cell survival.
Without sufficient levels of the protein, the motor neurons - which are nerve cells in the brain stem and spinal cord that control activities such as speaking, walking, breathing, and swallowing - die, leading to muscle weakness and atrophy.
There are four types of the condition.
These therapies are extraordinarily expensive as it is first of its kind gene modifying therapy, approved for rare neuromuscular disorders
Dr Sushil Garg,
NMC Royal Hospital
Type 1 is the most severe and many children with it do not live past the age of two. In July, The National reported on the case of three-month-old Malak Al Alami, who received Zolgensma after winning a global “lottery” run by the medicine’s manufacturer, Novartis. It is allocating up to 100 doses at no charge this year and has appointed a third party to select recipients every two weeks.
Children with Type 2 may sit without support, but cannot walk on their own. Symptoms typically appear between six and 18 months. Depending on the severity of their symptoms, children with this type may live a normal life span.
Type 3 is a milder form of the disease and resembles muscular dystrophy. Children are usually able to walk with difficulty and some eventually need a wheelchair. Symptoms usually appear around 18 months or in early childhood. They usually have a normal life expectancy.
Type 4 is very rare. It starts in young adulthood and results in mild motor impairment.
How does the treatment work and why is it so expensive?
Zolgensma is the only therapy designed to directly address the genetic root cause of the condition. It does this by replacing the function of the missing or nonworking gene, which is critical in making a protein essential to motor neuron cell survival.
It works by using a harmless, genetically engineered virus that travels around the body to help restore some of the protein missing as a result of the condition.
It is extremely effective, but hugely expensive because its market is very small and it required a lot of research to develop it, Dr Sushil Garg, a consultant neurologist at NMC Royal Hospital in Dubai, told The National.
“These therapies are extraordinarily expensive as it is first of its kind gene modifying therapy, approved for rare neuromuscular disorders,” said Dr Garg.
“Any medicine which is for gene modifying or work on [the] immune system has to undergo a lot of research and trials and approval processes, hence is pretty expensive.”
However, it currently is only used in children under two, but that may change in time.
New trial
Novartis launched a trial earlier this year to evaluate the safety and efficacy of the drug in children with SMA weighing between 8.5kg and 21kg.
The results will supplement emerging real-world evidence and use of the therapy in the European Union and Canada, where regulators have approved the drug for use in babies and young children up to 21kg.
Abu Dhabi residents Rahul Pathak, 37 and his wife Astha Muthoo, 35, hope it will help their six-year-old daughter, Arya Pathak, who suffers from the condition. The National covered her plight earlier this week.
The disease has forced her to spend weeks at a time in intensive care. Even a common cold can take a heavy toll on her weakened body.
Her parents cannot legally fundraise to help cover the cost of the drug, but donors can pay money directly to hospitals to help with medical bills.
Countries offering golden visas
UK
Innovator Founder Visa is aimed at those who can demonstrate relevant experience in business and sufficient investment funds to set up and scale up a new business in the UK. It offers permanent residence after three years.
Germany
Investing or establishing a business in Germany offers you a residence permit, which eventually leads to citizenship. The investment must meet an economic need and you have to have lived in Germany for five years to become a citizen.
Italy
The scheme is designed for foreign investors committed to making a significant contribution to the economy. Requires a minimum investment of €250,000 which can rise to €2 million.
Switzerland
Residence Programme offers residence to applicants and their families through economic contributions. The applicant must agree to pay an annual lump sum in tax.
Canada
Start-Up Visa Programme allows foreign entrepreneurs the opportunity to create a business in Canada and apply for permanent residence.
Mercer, the investment consulting arm of US services company Marsh & McLennan, expects its wealth division to at least double its assets under management (AUM) in the Middle East as wealth in the region continues to grow despite economic headwinds, a company official said.
Mercer Wealth, which globally has $160 billion in AUM, plans to boost its AUM in the region to $2-$3bn in the next 2-3 years from the present $1bn, said Yasir AbuShaban, a Dubai-based principal with Mercer Wealth.
“Within the next two to three years, we are looking at reaching $2 to $3 billion as a conservative estimate and we do see an opportunity to do so,” said Mr AbuShaban.
Mercer does not directly make investments, but allocates clients’ money they have discretion to, to professional asset managers. They also provide advice to clients.
“We have buying power. We can negotiate on their (client’s) behalf with asset managers to provide them lower fees than they otherwise would have to get on their own,” he added.
Mercer Wealth’s clients include sovereign wealth funds, family offices, and insurance companies among others.
From its office in Dubai, Mercer also looks after Africa, India and Turkey, where they also see opportunity for growth.
Wealth creation in Middle East and Africa (MEA) grew 8.5 per cent to $8.1 trillion last year from $7.5tn in 2015, higher than last year’s global average of 6 per cent and the second-highest growth in a region after Asia-Pacific which grew 9.9 per cent, according to consultancy Boston Consulting Group (BCG). In the region, where wealth grew just 1.9 per cent in 2015 compared with 2014, a pickup in oil prices has helped in wealth generation.
BCG is forecasting MEA wealth will rise to $12tn by 2021, growing at an annual average of 8 per cent.
Drivers of wealth generation in the region will be split evenly between new wealth creation and growth of performance of existing assets, according to BCG.
Another general trend in the region is clients’ looking for a comprehensive approach to investing, according to Mr AbuShaban.
“Institutional investors or some of the families are seeing a slowdown in the available capital they have to invest and in that sense they are looking at optimizing the way they manage their portfolios and making sure they are not investing haphazardly and different parts of their investment are working together,” said Mr AbuShaban.
Some clients also have a higher appetite for risk, given the low interest-rate environment that does not provide enough yield for some institutional investors. These clients are keen to invest in illiquid assets, such as private equity and infrastructure.
“What we have seen is a desire for higher returns in what has been a low-return environment specifically in various fixed income or bonds,” he said.
“In this environment, we have seen a de facto increase in the risk that clients are taking in things like illiquid investments, private equity investments, infrastructure and private debt, those kind of investments were higher illiquidity results in incrementally higher returns.”
The Abu Dhabi Investment Authority, one of the largest sovereign wealth funds, said in its 2016 report that has gradually increased its exposure in direct private equity and private credit transactions, mainly in Asian markets and especially in China and India. The authority’s private equity department focused on structured equities owing to “their defensive characteristics.”
Five films to watch
Castle in the Sky (1986)
Grave of the Fireflies (1988)
Only Yesterday (1991)
Pom Poki (1994)
The Tale of Princess Kaguya (2013)
How Beautiful this world is!
