WASHINGTON (Michigan News Source) – Those afflicted with Hemophilia B could receive a newly approved drug by the Food and Drug Administration (FDA), though they may be shocked by the $3.5 million per dose price tag. 

The drug, Hemgenix, has now assumed the title of the most expensive drug on the market to date, even surpassing the two most expensive drugs on the market, Skysona at $3 million to treat active cerebral adrenoleukodystrophy (CALD) and Zynteglo at $2.8 million per patient which regulates red blood cell transfusions. 

MORE NEWS: Four Years After Pandemic, Detroit Has Spent Under Half of Its American Rescue Plan Money

People with this rare condition experience a blood clotting disorder because of an insufficient amount of a protein named Protein IX.  The majority of those with Hemophilia B are men, though only 1 in 40,000 have the disorder. Women often are carriers and do not display symptoms but pass the disorder on to their children. 

At the moment, the best treatment is IV treatment for patients, though this is expensive and requires a regular and frequent schedule to yield best results.  Hemgenix is a one time dose via IV infusion that provides a gene therapy drug to patients for lasting change. 

Hemgenix consists of a viral vector carrying a gene for clotting Factor IX according to the FDA. The gene is expressed in the liver to produce Factor IX protein, to increase blood levels of Factor IX and thereby limit bleeding episodes. 

“Gene therapy for hemophilia has been on the horizon for more than two decades. Despite advancements in the treatment of hemophilia, the prevention and treatment of bleeding episodes can adversely impact individuals’ quality of life,”  Ph.D. and Director of the FDA’s Center for Biologics Evaluation and Research, Peter Marks said.  

Hemgenix’s maker, CSL Behring announced the drug’s price and effects once the FDA approved it. 

MORE NEWS: WMU Wins MAC Women’s Soccer Tournament

“In the ongoing clinical trial, HEMGENIX reduced the rate of annual bleeds and 94 percent of patients discontinued factor IX prophylaxis and remained prophylaxis-free,” the company said. 

The company’s chief executive officer commented on the unique role CSL has in the medical field at large. 

“As part of our promise to patients, CSL is committed to delivering innovative and groundbreaking solutions to address unmet medical needs, and we are proud to introduce the next wave of breakthrough medicines for people living with hemophilia B,” CSL’s Chief Executive Officer and Managing Director Paul Perreault said, “We recognize and thank all trial participants, scientists and investigators—without whom this important achievement would not have been possible—and look forward to seeing the positive impact of HEMGENIX on the hemophilia B community.”

According to the National Hemophilia Foundation, there are only eight other products licensed in the US to treat Hemophilia B.  CSL Behring intends to make Hemgenix available for eligible people with Hemophilia B as soon as possible according to a recent statement.  

Like most medicines in the U.S., most of the cost of the new treatment will be paid by insurers — not patients — including private plans and government programs according to WOOD-TV.