Thousands support Belgian family's fundraiser for world's most expensive medicine

Thousands support Belgian family's fundraiser for world's most expensive medicine
The family of 9-month-old Pia, who suffers from spinal muscular atrophy, are hoping to treat their toddler with the world's most expensive drug. Credit: TeamPia/Facebook

A fundraising campaign set up by a Flemish family whose toddler is fighting an aggressive and life-threatening genetic condition has seen Belgians respond en masse to their appeal.

The family of 9-month-old Pia set up a national SMS campaign in the hopes of raising enough money to obtain Zolgensma, a new gene therapy capable of curing spinal muscular atrophy.

The aggressive genetic condition progressively leads to muscular failure, and the prognosis for children is grim, with most not living past four years old.

Released this year, the medicine the family are hoping to obtain is also the most expensive medicine in the world, with a price tag for a single dose standing at €1.9 million.

"If one Belgian in ten sends a text message, the life of our daughter is saved," Pia's mother Ellen De Meyer told De Morgen.

The family's fundraising campaign, through which they receive €2 for every SMS sent out, has received widespread attention on mass and social media, leading to an "incredible" outpour of support the family.

"We now have more than €205,000, this is really incredible," De Meyer said, noting that despite the nearly €100,000 received through donations, they were still thousands of euros away from being able to afford the drug.

Beyond the price, the family's plight is further complicated by the fact that the drug has not yet been approved for the European market, meaning their daughter would need to be treated in the United States.

On Tuesday, the widely-circulated media reports of the campaign saw telecom operators Proximus and Telenet said they would stop withholding the €0.50 they normally retain with SMS campaigns.

The campaign also drew a response from the federal government, with the cabinet of Health Minister Maggie De Block saying that, until the drugs's months-long approval process is completed, they could not do anything for baby Pia.

The minister's cabinet added the family could continue to have access to Spinraza, an European market-approved medicine which costs €88,000 per spinal cord shot.

Spinraza, which treats but does not cure the disease, can be administered to toddlers up to 2 years old.

Gabriela Galindo

The Brussels Times

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