ROCKVILLE, MD, May 24, 2021— TeamedOn International, Inc., a biotechnology company dedicated to advancing gene therapies for rare diseases, including ophthalmic indications, today announced a successful receipt of Orphan Drug Designation for its XLRS gene therapy program. The ownership of this adeno-associated viral vector expressing human retinoschisin-1 gene for treatment of X-linked juvenile retinoschisis (XLRS) was transferred from AGTC by mutual agreement.
Benefits of orphan drug designation include: (1) 7-year marketing exclusivity after approval; (2) Tax credits for qualified clinical (in humans) testing; (3) Waiver of the Prescription Drug User Fee (currently at almost $3 million for a new drug); (4) Eligibility to receive regulatory assistance and guidance from the FDA in the design of an overall drug development plan.
Under the Orphan Drug Act, the FDA may grant orphan drug designation to a drug or biological product candidate intended to treat a rare disease or condition. A disease is defined as “rare” if it affects less than 200,000 people in the United States, or if the cost of developing a drug and making it available in the United States for such diseases will exceed any potential profits from its sale.