Inside STAT: Wary hemophilia patients say they’re willing to wait longer for a safe gene therapy

(ADOBE)

The FDA's rejection earlier this week of a hemophilia A drug that many in the industry thought was going to get a green light came as a surprise to patients as well — but many say they're willing to wait if it means companies can get it right. The drug, called Roctavian and developed by BioMarin, seemed to fix the genetic hiccup that leads to hemophilia, but its effect on an important clotting factor waned over time in studies. While the news of the rejection made for a sad day, “[I]t’s also a good day because I think this community has fought long and hard for safety to be a priority,” Michelle Rice, who has a mild case of the disease and has two sons with severe forms of hemophilia, tells STAT's Elizabeth Cooney. Read more here.