Galibra Neuroscience Receives FDA Orphan Drug and Rare Pediatric Disease Designations for SSADH Deficiency Gene Therapy

▤ Summary

Galibra Neuroscience announced that the FDA granted Orphan Drug Designation and Rare Pediatric Disease Designation for its investigational SSADH deficiency gene therapy. The company says the program aims to become the first disease-modifying therapy for this ultra-rare neurologic disorder. Galibra is still in the IND-enabling stage and plans to move toward first-in-human clinical trials with support from academic and patient advocacy partners. The article also highlights strategic clinical and regulatory support from Aurelix Bio and the SSADH patient community.

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