FDA Grants Rare Pediatric Disease Designation to ABO-202 for Infantile Batten Disease

The U.S. Food and Drug Administration (FDA) has granted a rare pediatric disease designation to Abeona Therapeutics’ investigational ABO-202 program being developed for Batten disease caused by mutations in the CLN1 gene, also known as infantile neuronal ceroid lipofuscinosis.

ABO-202 is an adeno-associated virus (AAV)-based gene therapy (AAV-CLN1) where weakened viruses are engineered to deliver a normal copy of the CLN1 gene to target cells.

Preclinical studies with animal models of infantile Batten disease showed that ABO-202 gene therapy, when administered early in the disease course as a single intrathecal (spinal cord) injection of self-complementary AAV9 encoding a healthy human CLN1 gene in pre-symptomatic CLN1 mice, significantly increased their survival, improved behavior, and reduced motor deficits.

Response to treatment improved with higher doses and a combination approach using both intravenous and intrathecal routes for ABO-202 delivery, further increased survival by 50 percent, suggesting the treatment might be of benefit in symptomatic animal models.

The FDA’s decision is preceded by an orphan drug designation for ABO-202 gene therapy granted in February 2018, which provides a special status for a drug or biological product to treat a rare disease, defined as those affecting fewer than 200,000 people in the U.S. The orphan drug designation grants a therapy, if approved, market exclusivity for up to seven years, as well as tax credits to cover the costs of qualified clinical testing, and other benefits.

The rare pediatric disease designation means that Abeona Therapeutics’ ABO-202 gene therapy, if approved for Batten disease, may receive a priority review voucher from the FDA, which then cab be used by the company for any drug to be given a priority review — a designation given only to a drug candidate that has demonstrated the potential to be a significant improvement in safety and effectiveness for a serious, unmet disease condition.

A priority review also means that upon receiving a Biologics License Application (BLA), the FDA compromises to review the application within six months after submission, compared to the standard 10 months.

“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,” Timothy J. Miller, PhD, president and CEO of Abeona Therapeutics Inc. said in a press release.

Clinical testing of ABO-202 may start later this year. “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,” he added.

Abeona Therapeutics also is developing another gene therapy, called AAV-based ABO-201, for children with juvenile Batten disease caused by mutations in the CLN3 gene. The FDA designated this gene therapy as an orphan drug in June 2017 as a potential therapy for that form of Batten disease.