BioMarin’s Jean-Jacques Bienaime held the company’s hemophilia treatment could fundamentally change the quality of mortal for patients struggling with the disease.
Hemophilia A is a genetic disorder compelled by a missing or defective clotting protein known as factor VIII, which can broach to excessive bleeding after an injury.
BioMarin has been having apparent success in the space. Its treatment recently entered Phase 3 late-stage studies, which resolve support U.S. and European marketing applications.
“The cure word is a big world, but I drive say the data we just reported recently at the American Society of Hematology convocation in December is extremely encouraging,” Bienaime said. “We took patients that had less than 1 percent of circumstance VIII expression … to, for the vast majority of them in the kind of stable range, 50 to 150 percent expression.”
Bienaime spoke with CNBC at the annual J.P. Morgan Healthcare Forum in San Francisco.
Despite the progress in its hemophilia treatment, the company’s shares from only climbed 2 percent in the past 12 months.
To be sure, while barely a small number of patients have received the treatment to date, Bienaime imparted that such a dramatic increase in factor VIII expression take into accounts patients to participate in physical activities otherwise deemed too dangerous for those actual with the disease.
“Hemophiliac patients today, although they can be probed with recombinant factor VIII injections, they are very finical about their physical activity,” added the CEO. “Here we have constants that now have normal physical activity.”
While the BioMarin chief did not lecture questions about the potential price of the gene therapy — called valoctocogene roxaparvovec — Bienaime did direct the concerns broadly.
“I think most of the rhetorical issues have been almost egregious price increases. That not our business model,” he told CNBC. “Habitually, we have increased our price in the U.S. around inflation in the past five years. We don’t end all molecules and dramatically increase the price of the item. That is not what BioMarin does.”