The Durability of Roctavian and the Need for Corticosteroids

Men with severe or moderately severe hemophilia B continue to display low bleeding rates and very little use of replacement therapies for at least three years following a single dose of the approved gene therapy hemgenix.

Briana Contreras, an editor with Managed Healthcare Executive, spoke with Harsha Rajasimha, MD, founder and executive chairman of IndoUSrare, in this month's episode of Tuning in to the C-Suite podcast. The conversation was about how the disparity in diversity and ethnicity in rare disease clinical trials in the U.S. has led to gaps in understanding diseases and conditions, jeopardizing universal health, and increasing the economic burden of healthcare.

Marstacimab is being reviewed to prevent or reduce the frequency of bleeding episodes in people with hemophilia A or B. The FDA has set an action date in the first quarter of 2024.

CTTP is rare hereditary blood clotting disorder stemming from a disease-causing mutation in the ADAMTS13 gene. This gene plays a crucial role in producing the ADAMTS13 enzyme, responsible for regulating blood clotting.

People with moderate or severe hemophilia had worse joint status, lower pain thresholds, and poorer quality of life compared with people with mild hemophilia or healthy controls.

Harsha Rajasimha, MD, founder and executive chairman of IndoUSrare, spoke to MHE about the current disparities in diversity and ethnicity in rare disease clinical trials in the U.S., as well as what he's seeing in clinical trials for diseases like hemophilia, in specific.