Monday, September 16, 2019
Akari Therapeutics, Plc, a biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where the complement (C5) and/or leukotriene (LTB4) systems are implicated, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for nomacopan for the treatment of bullous pemphigoid (BP).
“BP, a severe blistering skin condition with no approved treatments, is an exciting therapeutic target for our lead drug candidate, nomacopan. It is also a disease of increasing prevalence due to an aging population and improving diagnosis,” said Clive Richardson, Chief Executive Officer of Akari Therapeutics. “Orphan drug designation for nomacopan is a major step forward for the program, positioning nomacopan for eligibility for an additional seven years of marketing exclusivity in BP if nomacopan is approved by the FDA. This news is in addition to the recent orphan drug designation received for our HSCT-TMA program.”
The Company plans to release new safety and efficacy data from an ongoing Phase II trial with nomacopan in patients with BP at an oral presentation by Dr. Christian Sadik at the 28th European Academy of Dermatology and Venereology (EADV) Congress on October 10, 2019. In August, the Company announced new data demonstrating the synergistic benefits of nomacopan’s dual C5 and LTB4 inhibitory activity in pemphigoid disease, generated by Dr. Christian Sadik’s group at University of Lubeck, Germany, and published in the August 2019 edition of JCI Insight [link].
Orphan drug designation by the FDA is granted to promote the development of drugs that target conditions affecting 200,000 or fewer U.S. patients annually and that are expected to provide significant therapeutic advantage over existing treatments. Orphan designation qualifies Akari for various benefits, including seven years of market exclusivity following marketing approval, tax credits on U.S. clinical trials, eligibility for orphan drug grants, and a waiver of certain administrative fees.