FDA Grants Rare Pediatric Disease Designation to Sernova for Hemophilia A Program

Rare Daily Staff

The U.S. Food and Drug Administration has granted both Orphan Drug and Rare Pediatric Disease designations for Sernova’s hemophilia A program.

Hemophilia encompasses a group of inherited disorders that alter blood coagulation. Classical hemophilia, also known as hemophilia A, is a hereditary hemorrhagic disorder resulting from a deficit of FVIII that manifests as protracted and excessive bleeding either spontaneously or secondary to trauma. Hemophilia A is the most common form of hemophilia and is a genetic disorder caused by missing or defective FVIII, a blood clotting protein. Severe hemophilia A occurs in about 60 percent of cases where the deficiency of FVIII is less than 1 percent of normal blood concentration. While it is passed down from parents to children, about one third of cases are caused by a spontaneous change in the gene.

Hemophilia A occurs in about 1 in 5,000 births. Prolonged bleeding, in areas such as the brain, of a person with hemophilia A, can be fatal. Prolonged bleeding in joints can cause inflammatory responses and permanent joint damage. Approximately 20,000 people in the United States, 2,500 in Canada and 10,000 in Europe have moderate to severe forms of hemophilia A. Though there is no cure for the disease, hemophilia A can be controlled with regular infusions of recombinant clotting FVIII.

Sernova’s hemophilia A program combines the Sernova Cell Pouch with a patient’s own cells and will not require the use of immunosuppression medications. This therapy is intended to replace Factor VIII (FVIII)—an essential blood-clotting protein that is deficient or absent in patients with hemophilia A, which is accomplished by correcting the patient’s own blood outgrowth endothelial cells (BOECs) and subsequently returning them to the patient via the cell pouch. These modified cells function to release FVIII into the bloodstream, restoring the patient’s ability for clotting during periods of bleeding.

Sernova and research partners, through a Horizon 2020 grant which is part of the European Union’s research and innovation funding program in proof-of-concept work, successfully corrected human blood cells from patients with hemophilia A to produce Factor VIII using a novel first-in-class gene and cell therapy approach where the corrected cells were transplanted into the pre-implanted, vascularized Cell Pouch in a preclinical model of hemophilia A. The work demonstrated an improvement in blood clotting using the combined technologies.

The FDA grants orphan designation, also referred to as orphan status, to therapies intended for the treatment of rare diseases that affect fewer than 200,000 people in the United States. The designation provides certain benefits, including tax credits for qualified clinical testing, waiver or partial payment of FDA application fees and seven years of market exclusivity, if approved. Separately, rare pediatric disease designations are granted for rare diseases that primarily affect children under 18 years old with recipients of this designation being awarded a priority review voucher, upon approval. The priority review voucher may be redeemed, transferred, or sold. Most recently, Sarepta Therapeutics sold a PRV for $102 million.