This is the most expensive drug on the planet, but it could be the best option for patients.
FDA approved the first gene therapy to treat hemophilia B, a rare bleeding disorder. It is more expensive than any other drug.
The problem: Blood contains a protein called factor IX that aids in the formation of clots when vessels are damaged or cut. This reduces blood loss and internal bleeding after an injury.
Hemophilia B is caused by a genetic defect that stops them from making enough factor IX. Even a minor bump on the head can cause serious bleeding and even death.
People with hemophilia B will receive infusions of factor VIII every few days to avoid this situation. This varies depending on their treatment type. Although these infusions are lifesaving, they can also be costly, with an average cost of $274,000 annually in the US. What’s new? Hemgenix is now approved by the FDA. It is a hemophilia B treatment that CSL Behring owns. The engineered virus delivers a gene to produce factor IX to the liver. This allows hemophilia patients to make their own protein.
FDA evaluated a phase 3 trial in which 54 patients with severe or moderately severe hemophilia A received one injection of gene therapy. This classification indicates that their factor IX levels were below 2%. The normal range is between 50% and 150%. Six months after the gene therapy was administered, the average hemophilia level in participants was 39%. This placed them in the mild category. 18 months later, the levels were still at 37%. This suggests that the treatment is long-lasting (though it is not yet known how durable). FDA approved the first gene therapy to treat hemophilia B, a rare bleeding disorder. It is more expensive than any other drug.
The problem: Blood contains a protein called factor IX that aids in the formation of clots when vessels are damaged or cut. This reduces blood loss and internal bleeding after an injury.
Factor IX Infusions are life-saving but cost on average $274,000 annually in the US.
Hemophilia B is caused by a genetic defect that stops them from making enough factor IX. Even a minor bump on the head can cause serious bleeding and even death.
People with hemophilia B will receive infusions of factor VIII every few days to avoid this situation. This varies depending on their treatment type. Although these infusions are lifesaving, they can also be costly, with an average cost of $274,000 annually in the US. What’s new? Hemgenix is now approved by the FDA. It is a hemophilia B treatment that CSL Behring owns. The engineered virus delivers a gene to produce factor IX to the liver. This allows hemophilia patients to make their own protein.
FDA evaluated a phase 3 trial in which 54 patients with severe or moderately severe hemophilia A received one injection of gene therapy. This classification indicates that their factor IX levels were below 2%. The normal range is between 50% and 150%. 98% of the trial participants stopped receiving regular factor IX injections after the treatment.
Six months after the gene therapy was administered, the average hemophilia level in participants was 39%. This placed them in the mild category. 18 months later, the levels were still at 37%. This suggests that the treatment is long-lasting (although it is not yet clear how durable).
This resulted in 54% fewer bleeding events per annum compared to baseline and 98% of participants stopped receiving regular factor IX injections. Kim Phelan (COO) of the non-profit Coalition for Hemophilia B said, “We are delighted to witness this landmark in hemophilia B therapy.” She said, “Over the years, we have seen many advancements in hemophilia care, but gene therapy remains the first option to give those with hemophilia B and their caregivers the possibility of a life without the need for continuous, ongoing infusions.”
The cold water: This freedom is not cheap. A dose of CSL Behring’s hemophilia B treatment costs $3.5 million. However, hemophilia B patients could still expect to pay that much if infusions continue to be expensive. Depending on how durable Hemgenix is and the quality of their lives, $ 3.5 million could be a good deal.
Steven Pipe, trial leader, said that anecdotally the men who have undergone this treatment say they are enjoying not having to worry about hemophilia anymore.
“You are not doing infusions anymore. Your prophylactic therapy is not a part of your daily life.
