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Most expensive drug ever approved by FDA, a $3.5 million-per-dose gene therapy for hemophilia B

On Tuesday, U.S. health regulators cleared the first gene therapy for hemophilia. A one-time treatment costs $3.5 million for the blood-clotting disorder. The Food and Drug Administration (FDA) gave approval to Hemgenix, an IV treatment for adults with hemophilia B- the less common form of the genetic disorder which mostly affects men.


Right now, patients receive regularly, expensive IVs of a protein that facilitate the clotting of blood and stop bleeding.
The Pennsylvania based drugmaker CSL Behring, announced the $3.5 million price tag shortly after getting a green nod from FDA, saying its drug would ultimately decrease health care costs because patients would have fewer bleeding incidents and need fewer clotting treatments.

As per a study cited by the National Library of Medicine, the cost makes Hemgenix the most expensive medicine in the world, easily surpassing Novartis’ Zolgensma gene therapy for spinal muscular atrophy (SMA), which estimates about $2 million per dose and is also a single-dose medicine.

Mostly in the U.S., most of the cost of the new treatment is paid by the insurers, not patients. This covers private plans and government programs.

After years of research, gene therapies have begun reshaping cancer treatment and rare inheritable diseases with medicines that can improve or correct mutations implanted in people’s genetic code. Hemgenix is the first one of it’s kind treatment for hemophilia and several other drug making companies are researching on gene therapies for the more common form of the disorder, hemophilia A.

 FDA’s Dr. Peter Marks said,
“Today’s approval provides a new treatment option for patients with hemophilia B and represents important progress in the development of innovative therapies.”

However, the agency did not particularly mention how long the treatment works. But said that patients should benefit in terms of reduced bleeding and increased clotting for years.

The disease mostly strikes males and is caused due to mutations in the gene for a protein needed for blood clotting. Minor cuts or bruises can be life-threatening, and many people need treatments once or more a week to prevent fatal bleeding. If left untreated, hemophilia can cause bleeding that seeps into joints and internal organs, including the brain.

Hemgenix delivers a working gene for the clotting protein to the liver, where it is made.

Hemophilia B affects about 1 in 40,000 people and accounts for roughly 15% of those with the disease, according to the FDA.
The American regulatory authority FDA said it granted approval on the basis of two small studies, including one that showed those taking the drug had increased levels of the clotting protein, decreased need for standard treatment and a 54% decrease in bleeding problems.

Earlier this year, European regulators approved a similar gene therapy for hemophilia A. That drug, from drugmaker BioMarin, is still under review at the FDA.

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