A drug based on discoveries made at OMRF was honored today at the National Press Club in Washington, DC.
Each year, the National Organization for Rare Disorders honors individuals and companies for outstanding contributions toward improving the lives of people affected by rare diseases. This year, the group recognized Ceprotin, developed from the work of OMRF’s Charles Esmon, Ph.D., and Naomi Esmon, Ph.D.
Manufactured by Baxter Pharmaceuticals, Ceprotin combats a genetic deficiency of protein C. Left untreated, the deficiency causes blood clots that lead to amputations and ultimately death.
“It’s nice that this came to fruition,” said Charles Esmon, who holds the Lloyd Noble Chair in Cardiovascular Biology at OMRF. “We’re glad that Ceprotin has received this recognition, but we’re just happy knowing that it’s out there and available for people dealing with this genetic disorder.”
Ceprotin was the first drug approved by the European Union’s joint licensing protocol. In 2007, it received marketing in the U.S. from the Food and Drug Administration.
Genetic protein C deficiency affects about 1 in 300 people, according to Naomi Esmon. Insufficient protein C levels are first seen as blood clots in the blood vessels of the skin, eyes, brain and kidneys, but can occur anywhere in the body where blood flows.
“Without this drug, babies with protein C deficiency would die within the first week of life,” she said.