Is evolving research in complement inhibition a key to finding new medicines for rare diseases?

An uncharted pathway

Some of the greatest advancements in modern medicine started with a scientist taking a risk to boldly pursue new areas of research. For scientists at Alexion — a leading biotech company dedicated to treating rare diseases — that risk involved an extraordinary research journey to discover molecules that selectively target and inhibit the complement system, a part of the immune system that is essential to the body’s defense against infection.

In the beginning, this approach wasn’t widely understood or readily accepted. A belief in the science and a drive centered around patient safety enabled the company to move from a hypothesis to a potential medicine for a rare disease. The Alexion team’s vision was realized when the FDA approved the first complement inhibitor medicine in 2007 as a treatment for a single rare disease, but in many ways, the journey had just begun.

“Alexion’s success was born out of a unique experience — our team uncovered groundbreaking research that sparked a new way of understanding and approaching some rare diseases,” said Sharon Barr, Ph.D., Senior Vice President, Head of Research, Bioinformatics and Diagnostics at Alexion. “With compelling evidence that complement inhibition had the potential to treat one devastating disease, the team felt an immense responsibility to themselves, to other scientists in the field and most importantly to patients, to double down on its research efforts and seek out other diseases where complement inhibition might work too. We knew we had only scratched the surface.”

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