Rare Disease Report

BioMarin CEO Speaks on Gene Therapy for Hemophilia at J.P. Morgan

JANUARY 09, 2018
Mathew Shanley
At the annual J.P. Morgan Healthcare Conference in San Francisco, BioMarin Chief Executive Officer Jean-Jacques Bienaime spoke about the company’s development of valoctocogene roxaparvovec, a gene therapy in development for the treatment of Hemophilia A.

Formerly known as BMN 270, valoctocogene roxaparvovec was granted Breakthrough Therapy designation by the U.S. Food and Drug Administration on October 26. The special designation will allow the regulatory agency to expedite the development and review of the potential therapy, and was based on preliminary clinical evidence of the drug’s ability to provide “substantial improvement over existing therapies.”

Hemophilia A is a genetic disorder in which patient’s blood is unable to properly form clots as the result of reduced or completely absent levels of Factor VIII. Patients with this rare disorder experience recurrent painful bleeding episodes, some of which can be life-threatening.

While there are orphan drugs availabile to treat the bleeding episodes, and 2 orphan drugs – Advate and Kogenate – approved for prophylactic use in patients with hemophilia A, Bienaime believes that the company’s hemophilia treatment could “fundamentally change the quality of life” for patients living with the disease, as a gene therapy would, theoretically, eliminate they need for further prophylactic use of a drug.

"The cure word is a big world, but I would say the data we just reported recently at the American Society of Hematology meeting in December is extremely encouraging," Bienaime said to CNBC. "We took patients that had less than 1 percent of factor VIII expression ... to, for the vast majority of them, in the kind of normal range, 50 to 150 percent expression."

Such a dramatic increase in factor VIII expression would allow patients with hemophilia A to participate in physical activities otherwise deemed too dangerous.

"Hemophiliac patients today, although they can be treated with recombinant factor VIII injections, they are very careful about their physical activity," he added. "Here we have patients that now have normal physical activity."

For the full interview, watch below:


BioMarin CEO on hemophilia treatment success from CNBC.

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