The FDA has granted Rare Pediatric Disease Designation for the ABO-202 program (AAV-CLN1), an AAV-based gene therapy for the treatment of CLN1 disease (infantile and late infantile onset Batten disease). A fatal lysosomal storage disease of the nervous system caused by autosomal-recessive mutations in the CLN1 gene. In February 2018, the ABO-202 program was granted Orphan Drug Designation (ODD) by the FDA.
“This Rare Pediatric Disease designation for ABO-202 is a significant recognition of the strength of the data supporting a potential treatment for patients with CLN1, and is bolstered by the previous Orphan Drug designation from the FDA,” stated Timothy J. Miller, Ph.D., President & CEO of Abeona Therapeutics Inc. “These regulatory designations highlight the urgent need for a treatment for this devastating rare disease, and we look forward to initiating human clinical trials later this year.”
The rare pediatric disease designation indicates that the FDA may give the company a priority review voucher if the drug is approved for the rare pediatric indication. This is important because the priority review voucher can then be applied to another drug Abeona is developing or it can be transferred to another company.
Priority review vouchers are very valuable and have sold for as much as $350 million in the past. You can find more information about the priority review voucher program here. Abeona's most recent 10-Q showed a little over $56 million in cash on the balance sheet.