FDA Grants Fast Track Designation for Abeona’s Batten Disease Gene Therapy

June 18, 2019

The U.S. Food and Drug Administration granted fast track designation to Abeona Therapeutics for ABO-202, the company’s gene therapy in development for the treatment of infantile Batten disease (CLN1 disease), a fatal lysosomal storage disease.

Photo: João Siffert, CEO of Abeona

CLN1 disease typically manifests during the first year of life. It is caused by autosomal-recessive mutations in the CLN1 gene, which is responsible for production of the enzyme Palmitoyl protein thioesterase 1 (PPT1). A deficiency of PPT1 results in abnormal storage of proteins and lipids in neurons and other cells and impaired cellular function, causing symptoms including vision impairment/blindness, recurrent seizures, behavioral changes, and loss of motor skills. Most patients live into their twenties or thirties. There is currently no approved treatment available that can halt or reverse symptoms.

ABO-202 is an adeno-associated virus 9-based gene therapy that involves a one-time delivery of a normal copy of the PPT1 gene. In preclinical studies, ABO-202 normalized survival and improved neurological function in CLN1 mice. These studies also showed that a combination of intravenous and intrathecal administrations of ABO-202 improved efficacy over either delivery route alone, and that early treatment significantly improved outcomes.

Fast Track designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier through more frequent interactions with FDA, potential eligibility for accelerated approval, priority regulatory review, and rolling marketing application review. 

“Receiving Fast Track designation acknowledges the urgency for developing a therapy for children suffering from this rapidly-progressing and fatal disease and highlights the significant potential of ABO-202 to address this unmet need,” said João Siffert, CEO of Abeona.

Abeona also holds orphan drug designation for ABO-202 in the United States and European Union and rare pediatric disease designation for ABO-202 in the United States.

The company is preparing to initiate a phase 1/2 clinical trial evaluating ABO-202 in patients with CLN1 disease and said it will provide guidance on the timing of the trial later this year.

Author: Rare Daily Staff

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