The FDA has granted orphan drug designation to SIG-001, an investigational therapy under evaluation for hemophilia A, according to a press release from the drug’s manufacturer.
SIG-001 (Sigilon Therapeutics) uses Sigilon’s Shielded Living Therapeutics platform to implant cells that are engineered to produce stable blood plasma levels of factor VIII.
“We are very pleased to have received orphan drug designation for SIG-001. The designation underscores the critical unmet need for effective, durable therapies for hemophilia A and reinforces our commitment to advance SIG-001 through our development program,” Rogerio Vivaldi, MD, MBA, president and CEO of Sigilon, said in a press release. “This is the first of what we hope will be multiple orphan drug designations for Sigilon as we continue progressing novel therapies for chronic diseases through our pipeline.”
Preclinical studies have shown that Sigilon’s engineered cells — protected by Sigilon’s Afibromer biomaterials matrix, which prevents the immune system from rejecting the cells and avoids fibrosis — produce steady therapeutic levels of blood plasma clotting factor.
Clinical trials are expected begin in the first half of next year.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.