Hemgenix is the first gene therapy to treat adults with haemophilia B, a condition that affects approximately 40,000 people
THE United States Food and Drug Administration (FDA, its acronym in English) has approved a new treatment that costs about US$3.5 million (or R$18.6 million) per patient, making it the medicinal most expensive in the world. An independent study of the drug estimates a slightly lower price, at US$2.9 million (R$15.4 million).
Hemgenix is the first gene therapy to treat adults haemophilia ba disorder of genetic origin resulting from a lack of adequate levels of a clotting factor in the blood🇧🇷 Patients with this type of disease suffer from various bleeding, healing difficulties and joint pain.
According to the FDA itself, approximately 40,000 people, mostly men, currently have hemophilia B, which accounts for about 15% of all hemophilia cases.
In a study conducted by CSL Behring, the use of Hemgenix reduced the number of expected bleeding events throughout the year in patients by 54%. The need for patients to receive infusions also decreased by 94%.
THE gene therapy because hemophilia had been on the horizon for decades, according to Peter Marks, director of the FDA’s Center for Biological Evaluation and Research.
“Today’s approval provides a new treatment option for patients with hemophilia B and represents an important advance in the development of innovative therapies for those suffering a high disease burden associated with this form of hemophilia,” he told Insider.
A CSL spokesman said the company was confident the treatment would bring cost savings to the healthcare system. It would also help reduce annual rates of bleeding episodes and relieve the budget for prophylactic therapies, i.e. precautions to prevent bleeding.
The drug is expected to have a list price of $3.5 million, according to the Managed Healthcare Executive. An independent review by the Institute for Clinical and Economic Review said the fair price is about $2.9 million.
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Source: Terra
Camila Luna is a writer at Gossipify, where she covers the latest movies and television series. With a passion for all things entertainment, Camila brings her unique perspective to her writing and offers readers an inside look at the industry. Camila is a graduate from the University of California, Los Angeles (UCLA) with a degree in English and is also a avid movie watcher.
