FDA approves most expensive drug yet at $3.5 million per dose

CHICOPEE, Mass. (WWLP)- The FDA has just approved the most expensive drug on the market to date. The drug, known as Hemgenix, costs $3.5 million per dose and is administered to patients with the rare disorder Hemophilia B.

Hemophilia B is the rarer form of the blood clotting disorder resulting from insufficient amounts of a protein called Protein IX. Most patients with hemophilia B are men and about 1 in every 40,000 people has the disorder. Women are often carriers, displaying no symptoms but passing the disorder on to their children.

Small cuts or bruises can be life-threatening, and many people need treatments once or more a week to prevent serious bleeding. Left untreated, the condition can cause bleeding that seeps into joints and internal organs, including the brain.

Currently, patients receive frequent, expensive IVs of a protein that helps blood clot and prevent bleeding. Hemgenix is a gene therapy drug that is given in one dose via IV infusion.

“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,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. 

Drugmaker CSL Behring announced the $3.5 million price tag shortly after the FDA approval, saying its drug would ultimately reduce health care costs because patients would have fewer bleeding incidents and need fewer clotting treatments. The price appeared to exceed that of several other gene therapies priced upwards of $2 million.

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.

Famous people who have had hemophilia B include Alexei Nikolaevichm, the ill-fated only son of Russian Czar Nicholas II who was killed with the rest of his family in 1918 at the start of the Russian Revolution. Richard Burton, actor in movies like “Cleopatra” and “1984,” was one of the first actors to publicly reveal he had hemophilia.

The Associated Press contributed to this report.

You May Also Like

US downs Chinese balloon over ocean, moves to recover debris

(NewsNation) — The United States has downed a Chinese balloon off the…

University of Alabama initiatives honor retired Sen. Richard Shelby

TUSCALOOSA, Ala. (AP) — The University of Alabama on Friday announced two…

Democrats make South Carolina first presidential primary voting state

PHILADELPHIA — The Democratic National Committee on Saturday officially dethroned Iowa and…

New York school apologizes served watermelon and chicken waffles on first day of Black History Month

A food vendor in Rockland County, just outside of New York City,…

Off-duty NYPD officer shot and wounded while trying to buy car in Brooklyn, police say

An off-duty NYPD police officer trying to buy a car was shot…

Giant panda named Le Le dies after 20 years at Memphis Zoo

Giant panda Le Le has died, the Memphis Zoo announced Friday. The…

Defense Secretary Lloyd Austin's Statement on Downing of Chinese Balloon Is Ironic Comedy at Its Best

After a U.S Air Force F-22 Raptor shot down the Chinese surveillance…

Americans are tipping more often, even if they don’t want to

Americans may be grumbling about the new rules of tipping — whatever…

Meeting one of America’s few full-time custom knifemakers

Meeting one of America’s few full-time custom knifemakers – CBS News Watch…

Biden Finally Gets off His Duff, Comments on What Will Be Done With Chinese Spy Balloon

It looks like Joe Biden has finally gotten off his duff when…

Indian police arrest over 2,000 men for illegal child marriages

Indian police have arrested more than 2,000 men in a crackdown on…

Mo Bamba and Austin Rivers throw PUNCHES at each other and spark a huge bench-clearing brawl

Mo Bamba and Austin Rivers throw PUNCHES at each other and spark…