The world’s most expensive medicine—a one-time infusion that costs $3.5 million (28.6 crores) per dose—has been approved by US regulators for CSL Behring’s hemophilia B gene therapy. An inherited bleeding disorder is hemophilia B. Hemgenix works by introducing a gene capable of generating the missing clotting components into the liver, where it can produce Factor IX.
By treating the underlying causes of a variety of deadly illnesses, gene treatments can significantly improve them.
US regulators gave the hemophilia B gene therapy developed by CSL Behring the green light. This one-time infusion frees patients from ongoing treatment, but it is the costliest drug in the world at $3.5 million per dosage.
Hemgenix from CSL Behring, which is FDA-approved for treating bleeding disorders, reduced the number of bleeding events by 54% over a year in a study. Additionally, it relieved 94% of patients from undergoing time-consuming and expensive Factor IX infusions, which are presently required to manage the potentially fatal illness.
Brad Loncar, chief executive officer of Loncar Investments and a biotechnology investor, said, “While the price is a little higher than expected, I do think it has a chance of being successful because 1) existing drugs are also costly and 2) hemophilia patients constantly live in fear of bleeds.” Some people will find a gene therapy product appealing.
By treating the underlying causes of a variety of deadly illnesses, gene treatments can significantly improve them. When it was approved in 2019, Novartis AG’s Zolgensma for infants with spinal muscular atrophy cost $2.1 million, while Bluebird Bio Inc.’s Zynteglo for the blood condition beta-thalassemia cost $2.8 million.
Pricing has been a problem for innovative medications, with exorbitant prices for pharmaceuticals like Bluebird’s Zynteglo in Europe and Biogen Inc.’s Aduhelm for Alzheimer’s in the US contributing to their commercial failure.
The era of expensive, one-time miracle treatments is here:
Although there have been improvements in hemophilia treatment, Peter Marks, head of the US Food and Drug Administration’s Center for Biologics Evaluation and Research, warned that taking the necessary precautions to stop and manage bleeding can impair patients’ quality of life. According to him, Hemgenix marks a significant advancement in the creation of novel treatments for those affected by the condition.
The traditional medication for hemophilia involves injecting the body with the missing clotting factors so it can form clots and stop bleeding. Delivering a gene capable of producing the disappeared clotting components into the liver, where it begins to generate the Factor IX protein, is how Hemgenix works.
