The FDA approves the most expensive drug ever, a $3.5 million-per-dose gene therapy for hemophilia B
WASHINGTON — U.S. health officials on Tuesday approved the first gene therapy for hemophilia, a $3.5 million one-time treatment for the bleeding disorder. The Food and Drug Administration has approved Hemgenix, an IV treatment for adults with hemophilia B, the rarer form of the genetic disorder that primarily affects men.
Currently, patients receive frequent, expensive infusions of a protein that helps blood to clot and prevent bleeding.
Signage for CSL Behring, a unit of CSL Ltd., is seen in a July 2, 2014 file photo at one of the company’s plants in Melbourne, Australia. Carla Gottgens/Bloomberg via Getty
Pennsylvania-based drugmaker CSL Behring announced the $3.5 million price tag shortly after FDA approval, saying its drug would ultimately reduce healthcare costs because patients would bleed less and need fewer clotting treatments.
According to a study cited by the National Library of Medicine, the price makes Hemgenix the most expensive drug in the world, far surpassing Novartis’ Zolgensma gene therapy for spinal muscular atrophy (SMA), which costs around $2 million per dose and is also a single-dose Drug.
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As with most drugs in the US, most of the new treatment’s costs are borne by insurers rather than patients, including private plans and government programs.
After decades of research, gene therapies have begun to redesign the treatment of cancer and rare inherited diseases with drugs that can modify or correct mutations embedded in people’s genetic code. Hemgenix is the first such treatment for hemophilia, and several other drugmakers are working on gene therapies for the more common form of the disease, hemophilia A.
“Today’s approval offers a new treatment option for patients with hemophilia B and represents an important advance in the development of innovative therapies,” said Dr. Peter Marks from the FDA.
The agency did not specify how long the treatment would last. But CSL Behring said patients should benefit for years to come in terms of reduced bleeding and increased clotting.
Hemophilia almost always affects men and is caused by mutations in the gene for a protein needed for blood to clot. Small cuts or bruises can be life-threatening, and many people need treatment once or more a week to prevent serious bleeding. If left untreated, the condition can cause bleeding that invades 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 about 15% of those affected, according to the FDA.
The FDA said it granted approval based on two small studies, including one that showed those taking the drug had elevated levels of the clotting protein, reduced the need for standard treatments, and a 54% reduction in bleeding problems .
Earlier this year, European regulators approved a similar gene therapy for hemophilia A. This drug, made by drugmaker BioMarin, is still under FDA review.