Most expensive drug approved for hemophilia, at $3,500,000 per dose

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

The world’s most expensive drug was approved by the US Food and Drug Administration (FDA) this week 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 fail to make a 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 horizon for more than two decades. Despite advances in the treatment of hemophilia, the prevention and treatment of bleeding episodes can impact 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 offers a new treatment option for patients with hemophilia B and represents an important advance in the development of innovative therapies for those who suffer from the high burden of disease associated with this form of hemophilia.’

What is hemophilia B?

Hemophilia B is a genetic bleeding disorder that results from absent or insufficient levels of blood clotting factor IX, a protein needed for blood clots to form to stop bleeding.

Symptoms may include prolonged or heavy bleeding after an injury, surgery, or dental work; In severe cases, spontaneous bleeding episodes can occur without a clear cause.

Prolonged bleeding episodes can lead to serious complications such as: B. Bleeding into joints, muscles or internal organs, including the brain.

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

Treatment typically 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 levels of coagulation factor and prevent bleeding episodes.

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

“Hemgenix consists of a viral vector carrying a gene for coagulation factor IX. The gene is expressed in the liver to produce factor IX protein to increase blood levels of factor IX and thereby limit bleeding episodes,” the FDA said in a statement.

MORE : I’m a hemophiliac — I was afraid of bleeding for years until I got gene therapy

MORE: Cyclist Alex Dowsett on Hemophilia: ‘Real Victory Proves My Doctors Wrong’ Most expensive drug approved for hemophilia, at $3,500,000 per dose

Justin Scacco

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