Haemophilia, a rare genetic disorder characterised by insufficient production of a critical blood clotting protein in the liver, remains a condition with uncertain global prevalence, often eluding diagnosis. According to a 2019 study featured in the Annals of Internal Medicine, it is estimated that approximately 1,125,000 individuals worldwide—predominantly males—live with this hereditary bleeding ailment.
Even rarer is the subset of haemophilia patients, roughly 15%, who have haemophilia B, distinguished by a deficiency in the Factor IX clotting factor. Those afflicted with this variant endure persistent anxiety, fearing that even minor injuries or medical procedures might escalate into life-threatening bleeding episodes. Over time, their quality of life deteriorates due to limited mobility, joint damage, and excruciating pain. Until recently, the standard of care involved lifelong prophylactic treatment, involving regular infusions of Factor IX clotting factor.
A groundbreaking development in the field, CSL Behring's gene therapy known as HEMGENIX®, secured FDA approval in November 2022, promising to transform the management of this burdensome condition for patients and healthcare providers alike.
HEMGENIX, the outcome of two decades of rigorous research, stands as the sole FDA-approved gene therapy for haemophilia B. It not only offers substantial protection against bleeding compared to Factor IX prophylaxis but also holds the remarkable potential to enhance the quality of life for patients. What sets it apart is that this treatment is administered through a single infusion, marking a significant leap forward in the approach to managing haemophilia B.
Despite the potential of this revolutionary treatment, there exist significant obstacles along the path of patients considering gene therapy. Some individuals may exhibit reluctance to transition from a treatment regimen they find familiar and comfortable, while others may harbour reservations about gene therapy as a whole.
These challenges became evident when CSL Behring enlisted the support of BrightInsight. Drawing upon CSL Behring's extensive 30-plus years of service to the haemophilia community, their profound market expertise, as well as in-depth market research and a wealth of industry experts, both organisations meticulously examined the distinctive requirements of haemophilia B patients. In a collaborative effort, they proceeded to launch a comprehensive mobile companion application, not only tailored to patients utilising HEMGENIX but also catering to all patients undergoing Factor IX replacement therapy.
Introduced in April 2023, B SUPPORT stands as an all-encompassing mobile application that accommodates patients at every stage of their haemophilia B treatment journey. It offers a wide array of features, including customised educational resources, incident tracking capabilities, and improved communication channels with CSL Behring's support team, ensuring a holistic approach to patient care.
Click here to read the original news story.