Hungarians raise €2m for sick toddler to get world’s most expensive drug

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By Katherine Berjikian
Hungarians raise €2m for sick toddler to get world’s most expensive drug
Copyright  Facebook / Zente- SMA 1 Baby

A crowdfunding campaign in Hungary raised 700 million HUF (€2 million) in just a couple days for a sick toddler in Hungary to access the world’s most expensive drug.

Zente, 19 months, has a rare muscle disorder called spinal muscular atrophy (SMA) and requires a new treatment that his parents have labelled a "cure".

The drug, Zolgensma, must be administered before a child is two years old to be effective and costs $2.1 million (€1.9 million) for a single treatment.

Zente’s parents started a Gofundme page on September 19 to raise money for the drug.

On Wednesday, six days after they started the campaign, Zente’s mother announced that they had raised enough money for the treatment.

The World’s Most expensive drug

SMA is a group of neuromuscular disorders that result in the loss of motor neurons and progressive muscle wasting.

Zolgensma, which was released in May this year, is only available in the United States.

In a clinical trial of the drug, 36 children with SMA received the treatment before they were two and by the end of the trial, none of them needed a permanent ventilation system to breathe.

Zente’s parents are not the only ones trying to raise money for their child’s treatment — earlier this month, another family in Belgium raised a similar amount for the same drug.

One in 10 people in Belgium donated, raising a total of €1.9 million so a 9-month-old with SMA could access Zolgensma.

Some pharmaceutical companies in the US have called the cost of the drug "immoral", citing the fact that SMA is the leading genetic cause of death for infants.

There have been other concerns about the accuracy of the data its producer, Novartis, presented to the FDA before they approved the drug.

In response to criticism over the price of the drug Novartis has created a new "outcomes-based agreements" plan in which people only have to pay for the drug if it works.

It will also allow some insurance holders in the United States to pay for the drug over a five-year period.