Dual designations support development of a potential first disease-modifying therapy for an ultra-rare neurometabolic disorder

Galibra Neuroscience announced a significant regulatory milestone with the receipt of both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the U.S. 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. FDA-approved disease-modifying therapies for SSADH deficiency are absent; available treatment is focused primarily on symptom management.

The FDA grants ODD to drugs and biologics intended to treat rare diseases or conditions. The designation 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 similarly 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. The voucher may be transferred or redeemed to obtain priority review of a future marketing application.

Supported by compelling preclinical data obtained at Boston Children's Hospital and Harvard Medical School, Galibra's 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.

"Receiving both Orphan Drug and Rare Pediatric Disease Designations validates the importance of bringing innovative therapies to patients living with SSADH deficiency. These milestones reinforce our commitment to advancing a treatment that has the potential to address the underlying biology of this devastating disorder rather than simply managing its symptoms."

-Alexander Rotenberg, MD, PhD, Founder, Galibra Neuroscience

The Galibra program has been developed in close partnership with the SSADH Association and the broader international patient community, whose leadership has been instrumental in advancing natural history research, patient engagement, and therapeutic development. The recent ODD and RPDD designations are important milestones for individuals living with SSADH deficiency, their families, and the scientific community working to develop disease-modifying therapies.

"For families affected by SSADH deficiency, each milestone brings renewed hope. These designations represent another step toward a future in which treatment can address the underlying disease rather than solely managing symptoms. We are proud to partner with Galibra and to work together on behalf of individuals and families living with SSADH deficiency."

-Brad Hoffman, Founder and President, SSADH Association

Aurelix Bio provides strategic clinical development, regulatory planning, translational medicine, and operational support to accelerate advancement toward first-in-human studies.

"These FDA designations are meaningful regulatory milestones for the program and the SSADH community. They reflect the seriousness and rarity of SSADH deficiency and the importance of advancing innovative therapeutic approaches that target the underlying disease biology."

-Brandon M. Henry, MD, Aurelix Bio

Galibra remains focused on advancing its SSADH gene therapy program as rapidly as possible and looks forward to providing additional updates as development progresses.

About Galibra Neuroscience

Galibra Neuroscience is a biotechnology company developing transformative therapies for rare neurologic disorders. Through scientific innovation, rigorous development, and close collaboration with patient communities, Galibra advances programs designed to address the underlying biology of devastating genetic diseases with significant unmet medical need.

About Aurelix Bio

Aurelix Bio is a clinical development platform that accelerates rare disease therapies from concept to clinic. Aurelix partners with patient advocacy organizations, early-stage biotechnology companies, and academic investigators to navigate key translational inflection points, support strategic decision-making, and create disciplined development plans in resource-constrained settings.

SOURCE Galibra Neuroscience