Gene Therapies for Hemophilia Could Hit $1.5 Million, Analysts Speculate


As we get closer to seeing the U.S. Food and Drug Administration (FDA) green light the first gene therapy for hemophilia, analysts estimate that the cost of the probable one-time treatment could have a whopping price-tag of $1.5 million.

The predictions are primarily based on the current pricing of Spark Therapeutics’ recently-approved gene therapy treatment Luxturna, which costs about $425,000 for a treatment. Luxturna was the first gene therapy for eye disease approved by the FDA. Its approval marks the first time the FDA has approved a directly administered gene therapy that targets a disease caused by mutations in a specific gene.  

When Luxturna was approved earlier this year FDA Commissioner Scott Gottlieb said he believes gene therapy “will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses.”

Hemophilia could be one of those devastating illnesses that gene therapy can cure. In December Spark Therapeutics and its partner Pfizer showed off stunning interim data that showed a dramatic turnaround in some hemophilia patients. In the Phase I/II trial the patients were injected with SPK-9001, a therapy that helps hemophilia patients generate Factor IX, a blood-clotting protein missing in people with hemophilia B. Following a single treatment with SPK-9001 the presence of Factor IX activity in the body was 34 percent of normal. That is a huge change in the lives of the patients. As a result, annualized bleeding rate (ABR) was cut by 97 percent, from a mean rate of 11.1 events annually before treatment to 0.4 events per year after treatment. Factor IX concentrate use was cut by 99 percent, BioSpace reported at the time. Treatment for hemophilia B is repeated intravenous infusions of either plasma-derived or recombinant factor IX. If the clinical data holds up, SPK-9001 could be a veritable cure for the disease.

And that cure will have a cost. A Leering analyst estimated that the price for a gene therapy cure for hemophilia B will be about $1.5 million per patient. Leerink did note that the price could actually approach $2 million, but given uncertainties about responsiveness to the treatment, rebates and other pricing factors, the $1.5 million was considered to be a “reasonable estimate.” That is an increase in cost from the previously estimated $350,000 to $500,000.

“We believe the manufacturers are testing the waters currently as they embark on Phase 3 trials in order to determine where payors push back significantly. Based on our checks, a price somewhere in the range of $1-2M per patient seems most likely,” Leerink said in its note.

To keep things in perspective, Genentech’s approved hemophilia A clotting drug Hemlibra has a price tag of $425,000. During clinical trials, Hemlibra was shown to substantially reduce bleeds in adults and children who have hemophilia A with Factor VIII inhibitors. Hemlibra is not a one-and-done type of gene therapy. What the price will be for that kind of treatment is still to be determined. Companies like Spark and BioMarin will have to complete clinical studies and gain approval before a final cost will be determined.  

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