A drug developed by the lab of John Lambris, PhD, received orphan status in Europe this week for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare, life-threatening disease that causes anemia due to destruction of red blood cells and thrombosis. Orphan status brings such benefits as tax incentives, market exclusivity for 10 years, possibilities for additional research funding, and additional guidance from the European Medicines Agency during clinical development. This designation for the compound, called AMY-101, will allow Amyndas, the company currently developing the compound, to proceed with expedited clinical development. AMY-101 is a new way to fight PNH, which is currently only treatable with the most expensive drug available for sale in the United States. The new strategy is based on inhibiting C3, a central component of the oldest part of the human immune system – called “complement” – and could turn out to be less costly and more effective for the majority of patients with this rare blood disorder. (Note: Dr. Lambris developed AMY-101 at Penn and the university has licensed it to Amyndas, which is now further developing the compound for application in the clinic.)

Read the Department of Communications news release.