Hemophilia therapy could be first drug with $1M price tag: 3 things to know

Mackenzie Bean (Twitter) -

New gene therapies designed to cure hemophilia could cost $1.5 million or more, according to a May 7 Leerink analyst note cited by CNBC.

Here are three things to know.

1. The current treatment for hemophilia, which limits the blood's ability to clot, is factor replacement therapy. For adult patients, the treatment costs an estimated $580,000 to $800,000 a year, according to Leerink.

2. BioMarin, Spark Therapeutics and UniQure are all in the process of developing a gene therapy for hemophilia, which will likely have an even higher price tag, according to analysts.

"It appears the seemingly impervious million-dollar threshold may be breached with hemophilia gene therapy, which could do so while still creating value for society by reducing the cost of factor replacement therapy," Leerink analysts Joseph Schwartz and Dae Gon Ha wrote in the research note, according to CNBC.

3. Spark Therapeutics' Luxturna became the first gene therapy to win FDA approval Dec. 19. The therapy, intended to treat a rare form of blindness, received an $850,000 price tag, which Leerink analysts took into account when creating their price estimate for a hemophilia gene therapy.

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