There are few things more frightening and horrifying for a new parent than being told that their child has a lifelong medical disorder.
Perhaps the only words that could make this news more unsettling would be “crippling,” “incurable,” and “life-threatening.” Sadly, parents of children diagnosed with hemophilia are faced with those exact options.
Fortunately, options are available. And even more fortunately, the Food and Drug Administration (FDA) recently gave the distinction of “breakthrough” to a gene therapy being developed by UniQure.
Hemophilia B is a bleeding disorder where the clotting agent in the blood, known as Factor IX (FIX), is deficient. Without enough of the FIX in the blood, even small wounds can bleed for a prolonged period of time. More concerning is the possibility of internal bleeding, which may cause swelling and bruises, particularly in joints.
The FDA gives the “breakthrough” designation when the first clinical trials seem to show a major improvement over an existing therapy for a given condition. Some of the benefits of this status include priority review and special guidance from officials at the FDA. This status only came about in 2012, but it has not been without controversy.
The FDA already had mechanisms in place to expedite the approval process. These are “fast track,” “accelerated approval,” and “priority review.”
Perhaps more concerning is the fact that the word “breakthrough” seems to imply that the drugs picked to be a part of this program are miracles of some kind. The FDA recognizes that this is not the case, but public perception might matter more than intent or dictionary definitions.
Regardless, the new technology, called AMT-060, essentially uses tiny viruses to deliver a functional FIX gene to the individuals in order to boost the body’s natural ability to produce the missing clotting agent. These viruses are not harmful. In fact, these vectors have been used numerous times in the past as a means of delivering genes to the affected area.
With luck, this technology can reduce the number of people being affected by hemophilia B.
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