German authorities have cleared Cheshire drug maker Alexion Pharmaceuticals Inc. to formally open a clinical trial into using its treatment for a rare form of anemia to aid victims stricken in what is called the worst E. coli outbreak in history.
The Paul-Erhlich-Institut, Germany’s healthcare regulatory body, gave Alexion permission for a controlled study of its Soliris treatment on patients with Shiga-toxin producing E. coli hemolytic uremic syndrome (STEC-HUS).
Soliris is already approved in the U.S., Europe, much of Asia and Latin America, to treat patients who suffer a rare and potentially fatal anemia, called paroxysmal nocturnal hemoglobinuria, or PNH.
STEC-HUS resulted from infections from an especially virulent form of E. coli that broke out across Germany in May. It has sickened more than 3,400 people and killed 35.
On May 30, Alexion disclosed that it was donating thousands of doses of Soliris, also known as eculizumab, for use on some 800 confirmed cases of E. coli victims whose kidneys had been affected with STEC-HUS. The Soliris treatment can run into the hundreds of thousands of dollars a year per patient.
The German study is a response to critics concerned about the use of a drug not specifically approved to treat STEC-HUS symptoms.
German physician Rolf Stahl, M.D., lead investigator of the clinical trial, said  the study will “provide a framework for a coordinated and orderly collection of data to both educate physicians in the current crisis and to learn more about the potential of eculizumab to treat patients with STEC-HUS in the future.”
Investigators suspect the E. coli outbreak stems from ingestion of tainted produce.
