Aussie patient given new drug worth $5 million per dose
A potential cure for a rare genetic disease is being trialled, and it comes with a hefty price tag.
Sydney’s Royal Prince Alfred Hospital is part of a groundbreaking new trial aimed at potentially curing haemophilia B – a hereditary bleeding disorder.
Patients with haemophilia B have a mutation on the F9 gene, which causes excessive bleeding and damage to joins as they lack the protein that helps blood to clot.
A mere 40 patients worldwide will be infused with the gene therapy, with each dose the equivalent of $5 million, or over $1,000 a drop.
A safe virus carrying the F9 gene is infused into the body, sending it to the liver cells, where doctors hope the protein is made for the rest of the patient’s life.
Professor John Rasko, Head of Cell and Molecular Therapies at RPA, said the process is a “thrilling moment in the history of gene therapy”
"We are pushing the boundaries of medical technology hoping to bring cures to individuals," he said. "It's a massive international effort that takes time and money and ultimately the manufacturing alone costs millions and millions of dollars."
Haemophilia B patients currently require three injections a week. This revolutionary treatment could mean just one infusion in a lifetime.
The infusion on May 22 was the culmination of more than two decades of work.
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