Entrance to the FDA offices in Silver Spring, Maryland.AP
An innovative and effective drug, but at a stratospheric price. Investments by biotech companies in new gene therapies enable the development of drugs for diseases for which there was no adequate treatment. However, by influencing the price of the assumed costs, they make the new connections accessible to only a few. Hemgenix, a recently US-approved gene therapy for hemophilia, has just become the most expensive drug in history: $3.5 million. It is distributed by CSL Behring.
The new compound ousts Skysona from Bluebird Bio, a $3 million drug used to treat adrenoleukodystrophy, a type of genetic disease that damages the membrane that insulates nerve cells in the brain, as the most expensive drug. Zynteglo, another Bluebird Bio gene therapy for the blood disorder beta-thalassemia, cost $2.8 million earlier this year. Until this year, the most expensive drug was Zolgensma, a drug developed by Novartis’ gene therapy division and approved in 2019 to treat spinal muscular atrophy, a group of inherited muscle disorders that cause progressive muscle degeneration and weakness, at a price of 2, 1 million dollars.
What they all have in common is that they are enormously expensive, innovative gene therapies. CSL argues that despite its record price, its treatment can end up saving money because hemophilia B is a chronic disease for which conventional therapy is also very expensive and lasts indefinitely. Hemgenix is manufactured in Lexington, Massachusetts by uniQure NV, which sold the commercialization rights to CSL Behring in 2020. The high price makes it difficult for public health systems to finance the therapy. In the United States, it will only be available to billionaires or people with comprehensive health insurance coverage.
On November 22, the United States Food and Drug Administration (FDA) approved the marketing of Hemgenix (etranacogen dezaparvovec), an adeno-associated viral vector that, when injected, introduces the drug’s blood component into the body of people with hemophilia. The FDA has approved its use for the treatment of adults with hemophilia B (congenital deficiency of factor IX, a protein needed to form blood clots that stop bleeding) who are currently receiving prophylactic factor IX therapy or who are experiencing life-threatening bleeding have or have heavy and recurrent bleeding episodes spontaneously
majority of men
The majority of people with hemophilia B who experience symptoms are male. Symptoms may include prolonged or heavy bleeding after an injury, surgery, or dental work; in severe cases. Bleeding episodes can also occur spontaneously without a clear cause and, if prolonged, can lead to serious complications such as: B. Bleeding into joints, muscles or internal organs, including the brain. Hemophilia B affects about one in 40,000 people. About 16,000 people in the United States and Europe have it, according to uniQure. Hemophilia B accounts for about 15% of hemophiliacs.
Hemgenix is a single intravenous administration of a gene therapy product. Traditional treatment today usually consists of also giving substitutes for the missing or missing clotting factor intravenously to improve the body’s ability to stop bleeding and promote healing.
“CSL strives to provide innovative and pioneering solutions to address unmet medical needs, and we are proud to introduce the next wave of innovative medicines for people living with hemophilia B,” said Paul Perreault, CEO, in a statement from King of Prussia , the Pennsylvania-based biotech company CSL.
The safety and effectiveness of Hemgenix were evaluated in two clinical studies in men aged 18 to 75 with severe or moderate haemophilia B. Efficacy was measured by the decrease in annualized bleeding rate (ABR) in men. In a study involving 54 participants, subjects had elevated levels of factor IX activity, reduced need for routine factor IX replacement prophylaxis, and a 54 percent reduction in ABR compared to baseline.
“Gene therapy for hemophilia has been on the horizon for more than two decades. Despite advances in the treatment of hemophilia, the prevention and management of bleeding episodes can negatively impact people’s quality of life,” said Dr. Peter Marks, director of the Center for Hemophilia Evaluation, in a statement from FDA Biological Research. “The approval offers a new treatment option for patients with hemophilia B and represents an important advance in the development of innovative therapies for those with a high burden of disease associated with this form of hemophilia.”
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