The drug is a gene therapy used to treat adults with hemophilia B (Photo: Reuters/Andrew Kelly/File Photo)

The world’s most expensive drug was approved this week by the US Food and Drug Administration (FDA) for the treatment of hemophilia.

Hemgenix, made by CSL Behring, costs $3.5 million per treatment and is a gene therapy used to treat adults with hemophilia B, a genetic bleeding disorder in which people don’t produce the protein needed to form blood clots.

About 1 in 40,000 people suffer from the disease, most of whom are men.

Gene therapy for hemophilia has been on the agenda for more than two decades. Despite advances in the treatment of hemophilia, preventing and treating bleeding episodes can affect the quality of life of those affected,” said Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research.

“Today’s approval provides a new treatment option for patients with hemophilia B and represents an important advancement in the development of innovative therapies for people with high disease burden associated with this form of hemophilia.”

One in 40,000 has hemophilia B; that is about 15% of patients with haemophilia. Many female carriers of the disease have no symptoms. However, an estimated 10-25% of female carriers have mild symptoms and, in rare cases, moderate or severe symptoms.

Treatment usually involves replacing the missing or deficient clotting factor to improve the body’s ability to stop bleeding and promote healing.

Patients with severe hemophilia B typically require a routine regimen of intravenous (IV) infusions of factor IX substitutes to maintain adequate blood clotting factor levels and prevent bleeding.

Hemgenix is ​​a single dose gene therapy product administered as a single dose by intravenous infusion.

“Hemgenix consists of a viral vector that carries a gene for coagulation factor IX. The gene is expressed in the liver to produce factor IX protein, which increases blood levels of factor IX and thereby limits bleeding,” the FDA said in a statement.