Galibra Gains FDA Designations for SSADH Gene Therapy

Galibra Neuroscience has received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the US Food and Drug Administration (FDA) for its investigational gene therapy for succinic semialdehyde dehydrogenase (SSADH) deficiency.

SSADH deficiency is a rare inherited disorder caused by mutations in the ALDH5A1 gene, which disrupts GABA metabolism. 

The condition can result in intellectual disability, autism spectrum disorder, epilepsy and other neurological and psychiatric symptoms. 

There are currently no FDA-approved disease-modifying therapies for the disorder, with treatment mainly focused on symptom management.

The ODD provides development incentives for therapies targeting rare diseases, including potential tax credits, certain fee waivers and seven years of orphan drug exclusivity if the therapy is approved.
 
The RPDD is intended for treatments targeting serious rare diseases affecting children and may provide eligibility for a Rare Pediatric Disease Priority Review Voucher, subject to regulatory requirements.

Galibra is developing its gene therapy to address the underlying cause of SSADH deficiency.

Supported by preclinical research from Boston Children's Hospital and Harvard Medical School, the programme is progressing through IND-enabling activities.

The company is working with the SSADH Association, academic institutions and patient communities to prepare for clinical development and potential first-in-human studies.