FDA approves most expensive drug ever, a gene therapy for hemophilia B

FDA approves most expensive drug ever, a $3.5 million-per-dose gene therapy for hemophilia B

Nov 23, 2022 by CBS News

Key Facts

  • According to a study cited by the National Library of Medicine, the price makes Hemgenix the most expensive medicine in the world, easily topping Novartis' Zolgensma gene therapy for spinal muscular atrophy (SMA), which costs right around $2 million per dose and is also a single-dose medicine.
  • Hemgenix is the first such treatment for hemophilia and several other drugmakers are working on gene therapies for the more common form of the disorder, hemophilia A. "Today's approval provides a new treatment option for patients with hemophilia B and represents important progress in the development of innovative therapies," said the FDA's Dr. Peter Marks.
  • Hemophilia almost always strikes males and is caused by mutations in the gene for a protein needed for blood clotting.
  • The FDA said it granted approval based on two small studies, including one that showed those taking the drug had increased levels of the clotting protein, reduced need for standard treatment and a 54% drop in bleeding problems.

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