Rare Daily Staff

The U.S. Food and Drug Administration granted Abeona Therapeutics Rare Pediatric Disease Designation for ABO-202, an AAV-based gene therapy for the treatment of CLN1 disease, a form of the rare and fatal Batten disease.

CLN1 is a lysosomal storage disease of the nervous system caused by autosomal-recessive mutations in the CLN1 gene, also known as infantile neuronal ceroid lipofuscinosis. The genetic disease primarily affects the nervous system in newborns and progresses rapidly. Last month, the FDA granted ABO-202 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 Miller, president & CEO of Abeona. “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.”

Rare Pediatric Disease Designation makes a company eligible for a Rare Pediatric Disease Priority Review Voucher if the therapy granted the designation is approved. The designation was created to encourage the development of treatments for rare pediatric diseases. 

The vouchers are potentially lucrative because they are transferable and can be used to accelerate the review of large market therapeutics. The priority review voucher entitles the holder to priority review of a single New Drug Application or Biologics License Application.  Most recently Ultragenyx sold a rare pediatric priority review voucher it received for its MPSVII enzyme replacement therapy for $130 million.

March 15, 2018
Photo: Timothy Miller, president and CEO of Abeona