The U.S. Food and Drug Administration on Tuesday approved Baxalta Inc's Vonvendi, making it the first engineered protein-based treatment for the world's most common inherited bleeding disorder.
Vonvendi treats Willebrand disease, which is caused by the deficiency or defect in von Willebrand factor, a protein that is critical for blood clotting.
The disease affects about 1 percent of Americans and patients can develop severe bleeding from the nose, gums and intestines, as well as into muscles and joints.
Vonvendi, created through genetic engineering, is the first FDA-approved recombinant form of von Willebrand factor.
Baxalta, spun off from Baxter International Inc earlier this year, develops biotech treatments for rare blood conditions, cancers and immune system disorders.
The company owns Advate, which it says is the world's most prescribed hemophilia treatment.
UK-based Shire Plc has been rumored to be keen on buying Baxalta, which would help complement its growing portfolio of high-priced treatments for rare or "orphan" diseases.
Baxalta shares were up nearly 3 percent at $36.80 in late afternoon trading on Tuesday.