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Friday, February 3, 2023

The United States approved the most expensive drug in history: a new drug for gene therapy against hemophilia

WASHINGTON.—An innovative and effective drug, but at a stratospheric price. Investments by biotech companies in new gene therapies have made it possible to develop drugs for diseases that lacked adequate treatments. However, by passing the estimated cost on to the value, they convert the new compounds. only accessible to a few, Hemgenics, A hemophilia gene therapy Recently approved by the United States, it has just become the most expensive drug in history: 3.5 million dollars (about 3.3 million euros). It will be marketed by CSL Behring.

The new compound displaces Bluebird Bio’s Skysona, a $3 million drug to treat adrenoleukodystrophy, a type of gene condition that causes damage to the membrane insulating nerve cells in the brain, as the most expensive drug . Zynteglo, another Bluebird Bio gene therapy for the blood disorder beta-thalassemia, was valued at $2.8 million earlier this year. Until this year, the most expensive drug was Zolgensma, a drug developed by the gene therapy division of Novartis and approved in 2019 to treat spinal muscular atrophy, a group of inherited muscle diseases characterized by progressive muscle degeneration. and causes weakness, at a cost of 2.1 million dollars.

common to all of them Innovative Gene Therapy Extremely expensive CSL argues that despite its record price, its treatment can save money because hemophilia B is a chronic disease for which conventional therapy is too expensive and continues indefinitely. Hemgenix will be manufactured by Unicure NV in Lexington, Massachusetts, which sold marketing rights to CSL Behring in 2020. The high cost makes it difficult for public health systems to finance the therapy. in the United States It will be available only to billionaires or people with large coverage health insurance,

On November 22, the United States Drug Regulatory Agency (FDA) authorized the marketing of Hemgenix (etranacogene dezaparvovec), an adeno-associated viral vector that, when injected, introduces the blood component of the drug into the body. Is. Deficiency in 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 produce blood clots that stop bleeding), who currently require factor IX prophylactic therapy. are using, or who are presenting with life-threatening bleeding or bleeding episodes. severe and frequent spontaneous

Most people with hemophilia B who experience symptoms are men., Symptoms may include prolonged or heavy bleeding after an injury, surgery, or dental procedure; in extreme cases. Bleeding episodes can also occur spontaneously for no apparent reason and when prolonged can lead to serious complications, such as bleeding into the joints, muscles, or internal organs, including the brain. Hemophilia B affects about one in 40,000 people. According to Unicure, about 16,000 people in the United States and Europe have it. Hemophilia B accounts for approximately 15% of patients with hemophilia.

Hemgenics is a one-time intravenous administration of a gene therapy product. Conventional treatment now usually consists of the administration of replacement products, also intravenously, for the absent or deficient clotting factor to improve the body’s ability to stop bleeding and promote healing.

“CSL is committed to providing innovative and pioneering solutions to meet unmet medical needs, and we are proud to introduce the next wave of new medicines for people living with hemophilia B,” CEO Paul Perreault said in a statement. Feeling.” Pennsylvania-based biotech CSL.

The safety and efficacy of HemGenix was evaluated in two clinical trials in men 18 to 75 years of age with severe or moderately severe hemophilia B. Efficacy was measured by the reduction in annualized bleeding rate (ABR) for men. In one study, involving 54 participants, subjects had increased Factor IX activity levels, decreased need for routine Factor IX replacement prophylaxis, and a 54% decrease 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, prevention and treatment of bleeding episodes can negatively impact people’s quality of life,” Center for Hemophilia Evaluation Director of Dr. Peter Marks said in a statement. and FDA Biological Research. “The approval provides a new treatment option for patients with hemophilia B and represents significant progress in the development of innovative therapies for those who experience the high burden of disease associated with this form of hemophilia.”

by Miguel Jimenez

© EL Pace, SL


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