Belgian family hits fundraising target for worlds priciest drug

The family of a 9-month-old Belgian infant, Pia Boehnke, has raised almost €2 million in three days to pay for the spinal muscular atrophy drug Zolgensma, which has been approved in the U.S. but not in Europe, HLN reported Wednesday.

Pias mother appeared on Belgian television late Wednesday to thank “all of Belgium” for supporting their fundraiser.

Donors sent money through text message, with €2 donated for each message sent. The family will have to figure out whether to go to the U.S. or to France or Portugal to get the drug administered to Pia so that she can be treated for the deadly, muscle-destroying disease, her mother told HLN.

Zolgensma is administered through a one-time injection that targets the cause of the rare disease affecting Pia. While it is not a cure, it “improves muscle movement and function,” giving children with the disease a chance to live. Otherwise, they would likely die before the age of 2, according to the U.S. Food and Drug Administration, which approved the therapy earlier this year.

“We know very well that its not possible to fully cure her,” Pias mother, Ellen De Meyer, told Belgian broadcaster RTBF. “But a life in a wheelchair would be already very good.”

Pias case is very similar to that of the Portuguese baby Matilde, whose family managed this summer to raise money for Zolgensma. The Portuguese authorities paid for the drug in the end, local media reported.

Zolgensma, produced by Novartis subsidiary AveXis, is part of the latest generation of individualized therapies and carries a price tag of $2.1 million in the U.S. — which makes it the most expensive drug in the world. The gene therapy works by delivering a copy of a working gene that is dysfunctional in patients with spinal muscular atrophy (SMA).

The European Medicines Agency is due to issue a decision on approval before year-Read More – Source

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