The most expensive drug approved by FDA
In a groundbreaking development, the US Food and Drug Administration (FDA) has given its stamp of approval to the most expensive pharmaceutical breakthrough ever for the treatment of Hemophilia B.
This pioneering therapy was developed by the Australian company CSL Ltd. Hemophilia B is a rare and severe genetic condition characterized by an imbalance in the body’s ability to form stable blood clots due to a deficiency in clotting factor proteins.
As a result, individuals with Hemophilia B experience a heightened susceptibility to bleeding, even during routine daily activities. It predominantly affects males, but a small percentage of cases also afflict women who can potentially pass it on to their offspring through the X chromosome.
The newly approved drug, Hemgenix, comes with a staggering price tag of USD 3.5 million for a one-time treatment. While this cost may appear exorbitant, it is a justifiable investment when compared to the annual expenses associated with the conventional treatment, which involves weekly intravenous injections of factor IX. These injections typically range from USD 550,000 to 750,000 per annum.
Hemophilia B arises from a genetic deficiency of Factor IX, a crucial protein responsible for promoting clot formation in the body, thus halting bleeding. Patients with this condition often experience prolonged bleeding after injuries, dental procedures, or surgeries.
Traditional treatment involves regular intravenous infusions of Factor IX to enable the body to arrest bleeding. Hemgenix, on the other hand, takes a revolutionary approach by introducing a viral vector carrying a gene for clotting Factor IX. This innovation allows the patient’s own body to produce Factor IX protein, effectively increasing its levels and, consequently, reducing instances of bleeding.
Adverse reactions associated with Hemgenix are generally mild and include elevated liver enzymes, occasional headaches, and minor infusion-related responses. This groundbreaking drug promises to significantly improve the quality of life for individuals living with Hemophilia B, providing an innovative and potentially more cost-effective treatment option.
