Akari Therapeutics PLC said its medicine nomacopan received the orphan-drug designation in the U.S. to treat patients with complications associated with stem cell transplants.
The medicine is aimed at patients with hematopoietic stem cell transplant-associated thrombotic microangiopathy, or HSCT-TMA, which is a pattern of damage that can occur in the smallest blood vessels inside many of body's vital organs — most commonly the kidney and brain. These patients mostly have high platelet count, red blood cell fragments and high blood pressure.
The U.S. Food and Drug Administration's orphan-drug program is meant for medicines that can treat rare diseases or disorders that affect fewer than 200,000 people in the U.S. With the designation, Akari Therapeutics' medicine will be eligible for certain incentives, including tax credits for covering clinical trial expenses, prescription drug user fee waivers and seven-year market exclusivity after approval.
The London-based pharmaceutical company's drug has also received the fast-track designation in the U.S. for the same indication in children.
