People with one type of inherited blood illness hemophilia now have a $3.5 million one-time therapy option.
On November 22, the Food and Drug Administration authorized Hemgenix, a novel gene therapy. Soon later, CSL Behring, a pharmaceutical company, released the price.
According to the Associated Press, the business stated that their medicine would eventually lower healthcare expenses because people with the genetic disease would require fewer treatments for bleeding. As with the majority of authorized pharmaceuticals, private or government insurance, rather than patients, is expected to foot the expense.
Hemgenix is intended for people suffering from hemophilia B, a less prevalent variant of the condition. Patients are already given expensive IV treatments to increase coagulation and avoid hemorrhage. The therapy is also for patients who suffer severe spontaneous bleeding episodes regularly.
“Gene therapy for hemophilia has been on the horizon for more than two decades. Despite advancements in the treatment of hemophilia, the prevention and treatment of bleeding episodes can adversely impact individuals’ quality of life,” said the director of the FDA’s Center for Biologics Evaluation and Research Dr. Peter Marks,
“This approval provides a new treatment option for patients with hemophilia B and represents important progress in the development of innovative therapies for those experiencing a high burden of disease associated with this form of hemophilia,” Marks said in an FDA news release.
According to the FDA, people with hemophilia B have missing or inadequate quantities of blood clotting Factor IX, a protein required to build blood clots to halt bleeding.
Someone with this condition may endure prolonged or excessive bleeding following an accident, surgery, or dental procedure. Some people may have spontaneous bleeding without knowing why.
Hemophilia B is predominantly a male disease. Women can be carriers of the illness, but many do not exhibit symptoms. Approximately 10% to 25% of women carriers experience minor symptoms, while women might have moderate or severe symptoms in rare situations.
Hemophilia B affects roughly one in every 40,000 persons.
The disease can cause extended bleeding episodes as well as problems such as bleeding into joints, muscles, or internal organs such as the brain. Even minor wounds and bruises can be fatal.
Hemgenix is given as a single intravenous infusion. According to the FDA, it sends a functional gene for the clotting protein Factor IX to the liver, where it is produced.
Patients with hemophilia B who undergo conventional therapy receive monthly infusions of Factor IX replacement medications, which assist avoid bleeding episodes.
The novel gene treatment was tested for safety and efficacy in 57 men aged 18 to 75 with severe or moderately severe hemophilia B.
One trial with 54 participants found that the group had a lower requirement for routine Factor IX replacement and a 54% drop in the annualized bleeding rate.
Hemgenix side effects included increased liver enzymes, headache, minor infusion-related responses, and flu-like symptoms.
A comparable medication for those with hemophilia A, the more prevalent form of the disorder, was authorized by European authorities earlier this year. According to the Associated Press, the FDA is still studying that treatment.