Biotechnology research company Galibra Neuroscience announced on Wednesday the receipt of both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the US Food and Drug Administration (FDA) for the company's investigational gene therapy for succinic semialdehyde dehydrogenase (SSADH) deficiency.

SSADH deficiency is a rare inherited disorder of GABA metabolism associated with intellectual disability, autism spectrum disorder, epilepsy, and other neurologic and psychiatric manifestations. The syndrome is caused by pathologic variants in the ALDH5A1 gene, which disrupt normal GABA metabolism and lead to the accumulation of toxic neuroactive metabolites. Available treatment for SSADH is focused primarily on symptom management.

ODD is granted to drugs and biologics intended to treat rare diseases or conditions. It provides development incentives that may include tax credits for qualified clinical testing, waiver of certain FDA application user fees, and, if the therapy is approved for the designated indication, the potential for seven years of orphan drug exclusivity.

RPDD is available from the FDA for products intended to treat serious or life-threatening rare diseases that primarily affect children. If Galibra's therapy ultimately receives FDA approval and meets applicable statutory requirements and program timelines, the company may be eligible to receive a Rare Pediatric Disease Priority Review Voucher, which may be transferred or redeemed to obtain priority review of a future marketing application.

Galibra said that, supported by preclinical data obtained at Boston Children's Hospital and Harvard Medical School, its program is on track to be the first gene replacement therapy specifically designed to address the underlying cause of SSADH deficiency. Galibra is currently advancing IND-enabling activities for its SSADH deficiency program, and continues to work closely with academic and patient advocacy partners toward initiation of clinical trials.