There’s a new hope for people living with the rare blood disorder hemophilia B.
The FDA recently approved a new gene therapy treatment for those who suffer from the debilitating condition.
Dave Robinson, a 61-year-old IT consultant from Falls Church, Virginia, was born with the rare genetic blood disorder caused by an insufficient level of a protein known as factor IX.
That protein helps blood form clots to stop bleeding and seal wounds. Without it, hemophilia B patients bruise easily and bleed more frequently and for longer periods.
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“The biggest problems are caused by doing everyday things that will cause internal bleeding,” said Robinson.
Due to his condition, Robinson would give himself an infusion at home every 10 days. Things changed when he enrolled in a clinical trial and received a single infusion of BEQVEZ, a new Pfizer drug.
BEQVEZ is a one-time treatment that helps patients produce their own factor IX protein to prevent and control bleeding.
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“It’s very exciting. This provides us with an additional tool to treat hemophilia. Not all patients will be candidates for gene therapy, but for those that are good candidates, this is another game changer in their lifetime quality and also their ability to assume normal lives,” said Dr. Craig Kessler, a hematologist with MedStar Georgetown University Hospital.
“We will have to monitor these patients for at least 15 years to know if there’s durability to the gene therapy,” he said. “There are others. About 15% of individuals who don’t respond adequately to the gene therapy and in the clinical trials had to go back to their clotting factor concentrate regiments.”
Robinson took part in the trial seven years ago, eliminating his need for weekly infusions.
“I've been able to avoid treating myself 271 times … That burden has been completely removed,” he said.
Since the gene therapy, Robinson has pushed himself to walk 7,000 or 8,000 steps per day, and for the 7,000 Americans living with hemophilia B, that’s a huge accomplishment.
BEQVEZ is approved for those over the age of 18 who meet certain criteria, but doctors still aren’t sure what the out-of-pocket cost will be for patients.
