World’s most expensive medicine saves Vietnamese children from paralysis

When he was only three months old, Tuan Kiet’s parents brought him to the Vietnam National Hospital of Pediatrics in Hanoi after noticing symptoms of weak muscles.

By the time Kiet was admitted to the hospital in early 2021, his older brother was already paralyzed and in a wheelchair after being diagnosed with spinal muscular atrophy (SMA).

Kiet’s parents said the older brother, now 18 years old, could not sit by the time he turned eight months old.

The couple from Bac Ninh Province, which borders Hanoi, had their first-born son examined as an infant, but doctors only diagnosed him with a calcium deficiency. Several treatments did not improve his condition.

It was not until 2007, when the boy turned two, that he was finally diagnosed with SMA. Back then, there was no remedy for the disease. All the family could do was to have the boy do physical therapy to ease the symptoms. But eventually, he still ended up paralyzed.

Therefore, in late 2020, when their second child Kiet developed similar symptoms at an early age, they had him examined as soon as possible.

It turned out that Kiet also had SMA, a genetic disease that affects nerves and muscles, causing muscles to become increasingly weak. Those with SMA type 1, which is the most common type, often live for only two years.

But Kiet was luckier than his brother because in 2019 doctors started using a drug called Zolgensma to treat SMA.

The medicine is not a cure, but is designed to add a functional copy of a gene that is missing in babies born with SMA. It is applicable only to patients under two years old.

Costing US$2.1 million per dose, it was the most expensive medicine in the world in 2019.

In children with SMA, the SMN1 gene is missing or not working properly, and the drug, which is given as a one-time infusion into a vein, replaces the function of the missing or nonworking SMN1 gene with a new, working gene, according to the drug’s developer AveXis, which is owned by Novartis, a Swiss-American multinational pharmaceutical corporation.

The SMN1 gene is the body’s main source of SMN protein, and without it, motor neuron cells may not get the SMN protein they need to survive.

Motor neuron cells control muscle function. When motor neuron cells do not get enough SMN protein, they stop working. Once they are lost, they cannot be brought back. This means everyday activities like eating, breathing, or sitting up become harder to do, and can be lost permanently.

Doctor Nguyen Ngoc Khanh, deputy director of the Center of Endocrinology, Metabolism, Genetics and Molecular Therapy at the Vietnam National Hospital of Pediatrics in Hanoi, said Monday that given the cost, the medicine is only a dream for most SMA patients and their families.

But because the hospital has joined the Novartis’ Global Managed Access Program (gMAP), it is allowed to nominate one SMA patient for treatment to the drug corporation every two weeks for a lucky draw.

The gMAP provides Zolgensma free of charge to patients in countries where the therapy has not yet received approval, or in which no formal access pathway exists.

In 2021, the hospital won the lucky draw, and late that year, 11-month-old Kiet became the first patient in Vietnam to ever receive Zolgensma.

Vials of Zolgensma to treat spinal muscular atrophy. Photo courtesy of Vietnam National Hospital of Pediatrics in Hanoi.

According to the hospital, Zolgensma is currently only permitted in the U.S., Canada, Japan and the EU, and the journey to bring the drug to Vietnam and use it on patients “has gone through many complicated steps.”

As the drug’s shelf-life is only two weeks, doctors must ensure no problems during the process of transportation to get the drug in time.

“We had to make very careful calculations when injecting the medicine because with just one small mistake, the patient could be affected, or the drug will no longer be effective,” said Vu Chi Dung, director of the Center of Endocrinology, Metabolism, Genetics and Molecular Therapy.

Against all odds, Kiet successfully received Vietnam’s first dose of Zolgensma.

Half a year later, he could sit on his own and move his legs normally.

Since Kiet’s landmark dose, six other Vietnamese children have slowly beat SMA after receiving the unaffordable miracle injection at the hospital, also via joining the lucky draw and winning it.

Doctors at the institution are now waiting to obtain the medicine for 14 other child patients whose parents have registered for the treatment.

Doctor Khanh said the lottery is difficult to win because gMAP provides just 50 doses of the drug per year and many children need it.

Around the world, the lucky draw program and the therapy’s high cost has been criticized by more than a few.

According to a Guardian report in 2022, campaigning group Just Treatment likened the draw to a “real life Hunger Games,” while the charity group SMA Europe expressed alarm at the prospect of babies competing for lifesaving treatment.

Critics have previously criticized the staggering price of the drug, but the company says the cost is fair because it is a one-off treatment.