Big news for those with hemophilia!
On Thursday, the FDA approved Hemlibra (manufactured by Genentech), a weekly self-injection for hemophilia A patients who have developed resistance to standard medicines for preventing bleeding episodes.
Hemophilia A (also known as factor VIII deficiency or classic hemophilia) is an inherited genetic bleeding disorder caused by a lack of or defective clotting protein known as factor VIII (FVIII). Individuals with hemophilia A do not clot efficiently and experienced excessive and prolonged bleeding. Hemophilia A is mainly present in males. Females with hemophilia A are rare. It is estimated that hemophilia A occurs in approximately 1 in 5,000 births. There is estimated to be 20,000 people with hemophilia A in the U.S.
“Reducing the frequency or preventing bleeding episodes is an important part of disease management for patients with hemophilia,” Richard Pazdur, MD, acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research and director of the FDA’s Oncology Center of Excellence, said in a press release.
“Today’s approval provides a new preventative treatment that has been shown to significantly reduce the number of bleeding episodes in patients with hemophilia A with factor VIII inhibitors. In addition, patients treated with Hemlibra reported an improvement in their physical functioning.”
In the study, participants who got Hemlibra had a 79% reduction in the number of bleeding episodes compared to how they had previously fared on the older treatment.
The FDA noted the patients getting Hemlibra reported less pain, joint swelling and difficulty walking. Hemlibra does however carry the FDA’s strongest level of warning because it can cause severe blood clots when patients are given a certain other bleeding treatment at the same time.
This bit of treatment progress means more lives improved and strengthened with hope that they have more options for how to manage their rare disease!
What do you think about this latest approval? Share your stories, thoughts, and hopes, with the Patient Worthy community!
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