Hemophilia

Hemgenix is the first approved gene therapy in the US for hemophilia — and at $3.5 million per patient, it's also the world's most expensive drug.

In the late 1950s and much of the 1960s, fresh frozen plasma transfused in patients was the mainstay of hemophilia treatment. A gene therapy that provides lasting benefits would make that unnecessary, one of the reasons it has tantalized patients and scientists for 30 years. For his part, Wiseman has no regrets that he received the first approved gene therapy, for hemophilia B. About half of them in the United States eventually became infected through contaminated blood products, and thousands died, according to the National Hemophilia Foundation. Steven Pipe, a professor of pediatrics and pathology at the University of Michigan who served as principal investigator in the clinical trials for Hemgenix and Roctavian, said he has already spoken with two young men with hemophilia B who want to receive Hemgenix soon. The Talmud, a collection of Jewish rabbinical writings on laws and traditions, said baby boys did not have to be circumcised if two of their brothers bled to death from the procedure. But with them, he has run five marathons without fear of hemorrhages (his fastest time is three hours and 36 minutes), earned a master’s degree in public health, and plans to attend medical school in the fall. The FDA approved Hemgenix for males 18 and older; some of Croteau’s patients are as old as 24. “But I kind of approach it with the if-it-ain’t-broken-don’t-fix-it mentality. He said “multiple” insurers have pledged to cover the cost of the treatment but declined to name them. About 15 to 20 percent have hemophilia B while the rest have hemophilia A, which is caused by low levels of another protein known as factor VIII. Called Hemgenix, the gene therapy was cleared by the Food and Drug Administration in November, but has yet to be given to patients outside of a clinical trial.