Alxion Pharma receives FDA orphan drug designation for PNH
Release Date: 12-Jan-2017
Alexion Pharmaceuticals Inc. has recently announced that US Food and Drug Administration has granted orphan drug status to leading candidate ALXN 1210 for the treatment of patients suffering from paroxysmal nocturnal hemoglobinuria (PNH). PNH is a debilitating, ultra-rare, life-threatening blood disorder in which uncontrolled activation of complement, a component of the immune system, results in hemolysis (destruction of a patient’s red blood cells).
Alexion is currently enrolling patients in Phase 3 trials of ALXN1210 in patients with PNH as well as in patients with atypical hemolytic uremic syndrome (aHUS), another ultra-rare and life-threatening disease caused by chronic uncontrolled complement activation.
Orphan Drug Designation is granted by the FDA Office of Orphan Products Development (OOPD) to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven year period of U.S. marketing exclusivity, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and waiver of Prescription Drug User Fee Act (PDUFA) filing fees.
For Orphan Drug Clinical Insight Reports Contact: firstname.lastname@example.org
Need custom market research solution? We can help you with that too.
Kuick Research is a market research and analytics company that provides targeted information for critical decisions at business, product and
service levels. We are quick, predictive and known by the recommendations we have made in the past. Our result-oriented research methodology offers
understanding of multiple issues in a short period of time and gives us the capability to keep you full with loads of practical ideas.