$3.5 million ‘gene therapy’ for hemophilia gets FDA approval

WASHINGTON, Nov 23, (AP): US health regulators on Tuesday approved the first gene therapy for hemophilia, a $3.5 million one-time treatment for the blood-clotting disorder.

The Food and Drug Administration cleared Hemgenix, an IV treatment for adults with hemophilia B, the less common form of the genetic disorder which primarily affects men. Currently, patients receive frequent, expensive IVs of a protein that helps blood clot and prevent bleeding.

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 US, most of the cost of the new treatment will be paid by insurers — not patients — including private plans and government programs.

After decades of research, gene therapies have begun reshaping the treatment of cancers and rare inheritable diseases with medicines 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 disorder, hemophilia A..

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

The agency did not specify how long the treatment works. But CSL Behring said patients should benefit— in terms of reduced bleeding and increased clotting — for years.