Solid Biosciences has secured the US Food and Drug Administration (FDA) rare paediatric disease designation for SGT-212, its investigational gene therapy targeting Friedreich’s ataxia (FA).

The designation, which covers serious and life-threatening diseases affecting those under 18, may allow the company a paediatric priority review voucher for future regulatory submissions.

SGT-212 is designed to deliver the full-length human frataxin gene through a dual-administration route, combining direct intradentate nucleus (IDN) and intravenous (IV) infusions.

The frataxin gene will be delivered into the intradentate nucleus using a stereotactic, magnetic resonance imaging-guided device approved by the FDA.

The therapy aims to restore therapeutic frataxin protein levels to address FA's cardiac, neurological and systemic symptoms.

SGT-212 is a recombinant adeno-associated virus (AAV)-based gene replacement therapy for FA.

Solid Biosciences chief regulatory and preclinical operations officer Jessie Hanrahan stated: “Receiving paediatric rare disease designation marks another significant milestone for our Friedreich’s ataxia programme, SGT-212.

“Together with the fast track designation granted earlier this year, it recognises our dual-route clinical approach for FALCON, our first-in-human trial, which is now screening participants, as an important first step in meeting an unmet need for FA.

“These designations are designed to help accelerate time to market and enhance engagement with the FDA. We look forward to continued collaboration with regulators to bring this therapy to patients as quickly as possible.”

FA, a life-threatening, degenerative multisystem condition, results in progressive damage of the nervous system, then cardiac dysfunction and movement problems.

In January 2023, Solid Biosciences entered a strategic research collaboration with Phlox Therapeutics to expedite the development of new therapies for rare cardiac diseases.