With A Price Tag Of $3.5M Per Dose, The FDA Has Approved The Most Expensive Medication In The World.

Hemgenix is a one-time IV medication that can help hemophilia sufferers make half as many routine hospital visits, which will ultimately save health care expenses because there will be less bleeding.

The hemophilia B gene therapy developed by CSL Behring was given approval by the Food and Drug Administration. This one-time injection frees patients from ongoing treatment, but it is the most costly drug in the world at $3.5 million per dosage.

One dose of Hemgenix from CSL Behring reduced the anticipated number of bleeding events over the course of a year by 54%, according to a significant study on the medication. Additionally, it relieved 94% of patients from having to undergo 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 very expensive 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. A fair price for Hemgenix, according to an analysis by the nonprofit Institute for Clinical and Economic Review, would be between $2.93 million and $2.96 million.

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 significant advancement in the creation of novel treatments for those affected by the condition.

The traditional therapy for hemophilia involves injecting the body with the clotting factors it is missing so it can form clots and stop bleeding. Delivering a gene capable of producing the missing clotting components into the liver, where it begins to generate the Factor IX protein, is how Hemgenix works.
The commercialization rights for Hemgenix were sold to CSL Behring in 2020, and uniQure NV, which created the gene therapy, will produce it in Lexington, Massachusetts. Hemophilia B affects around 16,000 persons in the US and Europe, according to uniQure. About five times as many persons have hemophilia A, which is more prevalent.

Source: https://www.benefitspro.com/2022/11/28/fda-approves-worlds-most-expensive-drug-to-treat-hemophilia-at-3-5m-a-dose/