Experimental FA gene therapy targeting brain and heart earns EU orphan status

Solid Biosciences‘ experimental gene therapy for Friedreich’s ataxia (FA), called SGT-212, has been granted orphan drug status in the European Union.

This designation aims to offer additional incentives to companies investing in treatments for rare diseases, where the small number of patients can make it hard for drug developers to recoup development costs and turn a profit. The designation offers perks including reduced fees and a guaranteed 10 years of market exclusivity if the therapy is ultimately approved for use in the EU.

“For families affected by Friedreich’s ataxia, the need for new treatment options remains significant, and this designation supports the continued development of SGT-212 globally as we advance a therapy intentionally designed for the unique challenges of FA,” Jessie Hanrahan, PhD, chief regulatory and preclinical operations officer at Solid, said in a company press release.

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How SGT-212 targets FA

FA is caused by mutations in the FXN gene, which provides the biological instructions needed to produce a critical protein called frataxin.

When frataxin is deficient, mitochondria — cellular structures that are key to energy production — cannot function properly, leaving cells in the nervous system and in muscles, including the heart, without enough energy to function properly. This ultimately leads to FA symptoms.

SGT-212 is designed to deliver a healthy version of the FXN gene to cells in the brain and the heart. By restoring healthy frataxin levels, the therapy aims to normalize mitochondrial function, with the ultimate goal of easing symptoms and slowing disease progression.

The therapy is first administered directly into a brain region called the cerebellum, which is affected by FA, and then given by infusion into the bloodstream to target the heart and other affected tissues.

Solid is currently evaluating the therapy’s safety and early activity in an ongoing Phase 1/2 clinical trial called FALCON (NCT07180355), involving adults with FA. The first participant in the study was dosed a few months ago. Five participants have received the gene therapy so far, with no treatment-related serious safety issues reported. The trial continues to recruit eligible adults at clinical sites in Ohio, California, and Pennsylvania, and early results are expected next year.

“With five participants now dosed in the FALCON trial, we continue to build clinical experience with this first-in-class, dual-route approach and look forward to reporting initial clinical data in the first quarter of 2027,” Hanrahan said.