Abeona Receives FDA Rare Pediatric Disease Designation for ABO-202 Gene Therapy Program in CLN1 Dise

Abeona Therapeutics announced 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, also known as infantile neuronal ceroid lipofuscinosis, CLN1 disease is an inherited genetic disease that primarily affects the nervous system in newborns and progresses rapidly. 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," said 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. That voucher could then be used by the company for another drug—any drug—to be given a priority review. A priority review mandates that the FDA will review a BLA drug submission within 6 months instead of the standard 10 months. A priority review designation is only given to a drug candidate that has demonstrated the potential to be a significant improvement in safety and effectiveness for a serious, unmet disease condition. The priority review voucher may be used by the sponsor or transferred to accelerate the review timeline of another drug candidate.

Infantile and late infantile neuronal ceroid lipofuscinosis is a severe lysosomal disease caused by mutations in the CLN1 gene, which encodes the soluble lysosomal enzyme Palmitoyl-Protein-Thioesterase-1 (PPT1) and result in osmiophilic granules accumulating in lysosomes and leading to neuroinflammation, neurodegeneration and death. CLN1 disease is a neurodegenerative, manifests shortly after birth, and is fatal in its classic form by 6 to 12 years of age. In the classic form, aggressive clinical features appear, including rapid speech and motor deterioration, refractory epilepsy, ataxia, myoclonus, and visual failure between the ages of 6 and 24 months. By 5 years of age, CLN1 disease patients with the classic infantile form are typically poorly responsive and are no longer communicative. These patients subsequently die in the following few years.

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