Rare Pediatric Disease Designation Granted to Infantile Batten Disease Gene Therapy

This morning, Abeona Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to ABO-202, a gene therapy in development for the treatment of infantile and late infantile-onset Batten disease (CLN1 disease).

Orphan drug designation was granted to the ABO-202 program in mid-February.

The adeno-associated viral (AAV)-based gene therapy is intended for a one-time delivery of a normal copy of the defective CLN1 gene, which is responsible for production of the enzyme Palmitoyl protein thioesterase (PPT1).

In patients with Infantile neuronal ceroid lipofuscinosis (INCL), or infantile Batten disease, a deficiency of PPT1 results in abnormal storage of proteins and lipids in neurons and other cells, as well as impaired cellular function. Both can lead to symptoms including rapid speech and motor deterioration, refractory epilepsy, ataxia, myoclonus, and visual failure.

“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. in a press release. “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.”

Preclinical data collected from CLN1 mice exhibited the ability of a single intrathecal (IT) injection of self-complementary AAV 9 (scAAV9) encoding the human CLN1 gene to pre-symptomatic CLN1 mice at 1 week and 1 month considerably increased survival, improved behavior, and reduced motor deficits.

Rare pediatric disease 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 condition. Should ABO-202 be approved for the indication, the rare pediatric disease designation permits Abeona to receive a priority review voucher, which can be used by the company for another drug to be given a priority review. A priority review mandates that the FDA will review a biologics license application (BLA) drug submission within 6 months instead of 10 months.

The priority review voucher may be used by the sponsor or transferred to another company to expedite the review timeline of a different drug candidate. In February 2017, Sarepta sold the priority review voucher it was granted for its Duchenne muscular dystrophy (DMD) treatment eteplirsen (Exondys 51). Gilead paid $125 million for it.

In addition to ABO-202, Abeona is also developing ABO-201, a gene therapy intended for the treatment of juvenile Batten disease, which was granted orphan drug designation last summer.

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