There鈥檚 a new treatment now for thousands of patients in the U.S. who live with a rare disorder where blood doesn鈥檛 clot. The Food and Drug Administration recently approved Hemlibra, which is produced by Genentech, Inc.
Dr. Amy Shapiro is CEO of the in Indianapolis.
After eight years of development, the center took part in a clinical trial that lead to the FDA approving the new treatment for patients with Hemophilia A.
鈥淚鈥檝e been treating hemophilia and bleeding disorders for 35 years of my life, and I鈥檓 really excited about this,鈥� said Shapiro. 鈥淥ur patients are excited about this. Our staff is excited about this.鈥�
For the last 20 years, people with hemophilia A had to give themselves an injection, sometimes directly into a vein to clot their blood. Hemlibra allows doctors to place a clotting assistant underneath the skin, the same way diabetes medication is given.
鈥淚t鈥檚 now a subcutaneous injection. Anytime of the day that you give it is fine,鈥� said Shapiro. 鈥淵ou can go on once a week, or you can go on dosing once every four weeks.鈥�
Shapiro says it鈥檚 particularly beneficial for parents of young children.
鈥淔or families with children, getting them off to school, trying to get them infused before they go to school in the morning, that鈥檚 hard,鈥� said Shapiro. 鈥淭his is much easier. So this transforms their life.鈥�
Most cases of hemophilia are hereditary, but that鈥檚 not always the case. Shapiro says 30 percent of new cases are caused by a new genetic mutation.
Hemophilia A is the most common type of hemophilia, and according to Shapiro up to 450 patients in Indiana could benefit from the new treatment.
This story was produced by , a news collaborative covering public health.
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