CSL Behring’s Hemophilia Gene Therapy Approved
CSL Behring has won FDA approval for Hemgenix (etranacogene dezaparvovec-drlb), a gene therapy with the potential to cure hemophilia B and — at $3.5 million per treatment — the most expensive drug in the world.
While the price tag may make insurance companies cringe, it pales in comparison to the lifetime costs of the disease it treats, a CSL spokesperson told FDAnews. “The costs to the healthcare system of treating people with moderate to severe hemophilia B can be significant over a lifetime — by some estimates more than $20 million per person,” CSL Behring said.
The Institute for Clinical and Economic Review concluded that Hemgenix could save the overall healthcare system $5 to $5.8 million per person. The same review determined that Hemgenix would be cost-effective if priced at $2.9 million.