Steven Pipe's patients think they are cured. They received a one-time gene therapy in a trial called Pipe that was meant to be used for a specific type of bleeding disorder. Gene therapy addresses the underlying cause of a disease. They didn't have to worry about bleeding for years thanks to the treatment.
The hematologist at the University of Michigan Health System says that people don't have to think about their bleeding disorders anymore. This looks like a cure for a lot of things.
The therapy, called Hemgenix, gained US approval from the Food and Drug Administration. It costs the most in the world. The trial participants didn't have to pay for the therapy. Future patients will.
Some patients who were dosed in earlier trials are still keeping bleeds to a minimum. Gene therapy experts are not surprised by the price. It's reasonable and expected. Nicole Paulk is an assistant professor at the University of California, San Francisco.
The approval is a big deal for patients. Many patients have to avoid sports and hobbies because of the pain and mobility issues caused by the repeated bleeding of the disease. Bleeding can be small and painless. It could be a problem. Bleeding in the joints can cause arthritis, and it can be fatal if it gets into the brain. A patient's life can be changed by preventing bleeds.
The cause of hemophilia B is a genetic abnormality. Factor IX is needed to make blood clot. Patients need to get regular IV injections of Factor IX. It has to be administered at least once a week because of the short amount of time it takes for the molecule to reach the bloodstream. A patient with severe hemophilia B may need to get injections of the clotting factor concentrate two or three times a week for the rest of their lives. The burden is a significant one.
According to an analysis in The American Journal of Managed Care, these injections cost patients an average of $127,194 annually. According to a study in the Journal of Medical Economics, treatment for severe and moderately severe bleeding disorders can cost $300,000 or more.
All Rights Reserved © 2024
