Sernova Gets FDA Orphan Drug, Rare Pediatric Disease Designations For Hemophilia A ProgramNovember 27, 2023
Sernova Corp. (SVA.TO, SEOVF), a clinical-stage biotechnology company focused on cell therapeutics, announced Monday that the U.S. Food and Drug Administration has granted both Orphan Drug Designation or ODD and Rare Pediatric Disease Designation or RPDD for its Hemophilia A program.
The company further announced a research collaboration with the University of Piemonte Orientale, Italy under the direction of Antonia Follenzi, Professor of Histology and Cell and Gene therapy.
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. The therapy is intended to replace Factor VIII (FVIII) – an essential blood-clotting protein that is deficient or absent in patients with Hemophilia A.
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 U.S.
Cynthia Pussinen, Chief Executive Officer of Sernova, said, “We are pleased with the FDA’s decision to grant these designations for our novel treatment for Hemophilia A, which uses the Cell Pouch in combination with cells corrected for the production of Factor VIII. Hemophilia A is a serious, life limiting condition and we are committed to advancing development of the program, with a hope to positively impact patients around the world who are waiting for improved treatments.”
According to the company, the combined benefits of these ODD and RPDD designations include exclusive marketing rights for a seven-year period, after marketing approval, a 25% federal tax credit for clinical research expenses incurred in the U.S. which is applicable for up to 20 years, and waiver of Prescription Drug User Fee Act (PDUFA) fees for orphan drugs, among other things.
Once the therapy is approved for marketing, it cannot be copied and sold in the U.S. for 7 years regardless of patent life and the sponsor will be granted a Priority Voucher which can be used to receive approximately 4 months reduction time of the standard FDA review period or sold.
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