Solid Biosciences receives FDA rare pediatric disease designation for Friedreich’s Ataxia Gene Therapy SGT-212

USA—Solid Biosciences has received rare pediatric disease designation from the US Food and Drug Administration (FDA) for SGT-212, an investigational gene therapy targeting Friedreich’s ataxia (FA).

This designation applies to serious and life-threatening diseases that affect children under 18 years old.

The status may qualify the company for a pediatric priority review voucher when it submits future regulatory applications.

The therapy delivers the full-length human frataxin gene using a dual-administration approach that combines direct intradentate nucleus (IDN) injection with intravenous (IV) infusion.

Medical professionals administer the gene into the intradentate nucleus using an FDA-approved stereotactic device guided by magnetic resonance imaging.

This targeted delivery method aims to restore therapeutic frataxin protein levels throughout the body, addressing the cardiac, neurological, and systemic symptoms associated with FA.

SGT-212 employs recombinant adeno-associated virus (AAV) technology as a gene replacement therapy.

The treatment seeks to compensate for the genetic deficiency that causes Friedreich’s ataxia by introducing a functional copy of the frataxin gene into patients’ cells.

Jessie Hanrahan, Solid Biosciences’ chief regulatory and preclinical operations officer, described the designation as a significant milestone for the company’s Friedreich’s ataxia program.

She noted that the FDA granted fast track designation to SGT-212 earlier this year, recognizing the dual-route clinical approach used in FALCON, the company’s first-in-human trial that has begun screening participants.

Hanrahan emphasized that these designations help accelerate the path to market while enhancing engagement with regulatory authorities.

She expressed the company’s commitment to working with regulators to deliver the therapy to patients as quickly as possible.

Friedreich’s ataxia represents a life-threatening, degenerative condition that affects multiple body systems.

The disease causes progressive damage to the nervous system, leading to cardiac dysfunction and movement difficulties over time.

Patients with FA face declining physical abilities as the condition advances, making effective treatments a critical unmet medical need.

In January 2023, Solid Biosciences formed a strategic research collaboration with Phlox Therapeutics to accelerate the development of new treatments for rare cardiac diseases.

This partnership supports the company’s broader efforts to address conditions like Friedreich’s ataxia that involve significant cardiac complications alongside other systemic effects.