Solid Biosciences gets FDA rare pediatric disease nod for Friedreich’s ataxia gene therapy

The U.S. FDA has granted rare pediatric disease designation to Solid Biosciences’ investigational gene therapy for Friedreich’s ataxia (FA).

The rare pediatric disease designation provides Solid with the potential to receive a pediatric priority review voucher (PRV), which can expedite the review for future BLAs or be sold to another company.

SGT-212, a recombinant AAV-based gene replacement therapy, is designed to deliver the full-length frataxin gene via dual routes of administration, utilizing both direct intradentate nucleus and intravenous infusions, with the goal of promoting restoration of therapeutic levels of the frataxin protein to address neurologic, cardiac and systemic clinical manifestations of FA.

FA is an inherited, life-threatening, degenerative multisystem disease caused by defects in the frataxin gene that disrupt production of the frataxin protein, a mitochondrial iron-binding protein involved in essential cellular processes, including energy production. FA is known to cause progressive nervous system damage, movement problems, and cardiac dysfunction, with cardiac complications identified as the primary cause of death.