US health regulators this week approved the first gene therapy for hemophilia, a one-time, $3,5 million treatment for the blood-clotting disorder.
Η Υπηρεσία Τροφίμων και Φαρμάκων (Food and Drug Administration ή FDA) ενέκρινε το Hemgenix, μια ενδοφλέβια θεραπεία για ενήλικες με αιμορροφιλία Β, τη λιγότερο κοινή μορφή μιας γενετικής διαταραχής που επηρεάζει κυρίως τους άνδρες. Αυτή την στιγμή, οι ασθενείς λαμβάνουν συχνές, δαπανηρές ενδοφλέβιες δόσεις μιας πρωτεΐνης που βοηθά στην πήξη του αίματος και στην πρόληψη της αιμορραγίας.
Pennsylvania-based pharmaceutical company CSL Behring announced the $3,5 million price tag shortly after the FDA approval, saying its drug will ultimately lower health care costs because patients will have fewer bleeding events and fewer doses will be needed for blood clotting.
According to a study by the National Library of Medicine, the price makes Hemgenix the most expensive drug in the world, easily surpassing Novartis' Zolgensma gene therapy for spinal muscular atrophy (SMA), which costs about $2 million per dose and is also single-dose medicine.
Like most drugs in the US, most of the cost of the new treatment will be paid by private or government insurance companies, not patients.
After decades of research, gene therapies have begun to reshape the treatment of cancers and rare inherited diseases with drugs that can modify or correct mutations built into people's genetic code.
Hemgenix is a first such treatment for hemophilia. Several scientists are working on gene therapies for the most common form of the disorder, hemophilia A.
