FDA awards new status to SGT-212 gene therapy for FA kids, adults
Rare pediatric disease designation helps incentivize treatment development
SGT-212, a gene therapy candidate for Friedreich’s ataxia (FA) that’s currently in early clinical testing, has been granted rare pediatric disease designation by the U.S. Food and Drug Administration (FDA).
The FDA awards this status to experimental treatments that are designed to address rare diseases that primarily affect children, with the aim of incentivizing companies to develop such therapies.
With this designation, if SGT-212 ultimately wins regulatory approval, its developer, Solid Biosciences, would get a voucher that can be redeemed to secure faster FDA review of a different experimental therapy. Solid could either use the voucher itself or sell it to another company.
The new rare pediatric disease status is the second FDA designation that SGT-212 has received this year. Back in January, the gene therapy was granted fast track designation, which aims to speed the development of experimental medicines that have the potential to address unmet needs in the care of serious conditions.
“Receiving pediatric rare disease designation marks another significant milestone for our Friedreich’s ataxia program, SGT-212,” Jessie Hanrahan, PhD, Solid’s chief regulatory and preclinical operations officer, said in a company press release. “Together with the Fast Track designation granted earlier this year, it recognizes 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.”
Friedreich’s ataxia is caused by mutations in the gene that encodes frataxin, a protein that’s essential for the proper functioning of energy-generating cellular structures called mitochondria. Lack of functional frataxin leads to mitochondrial dysfunction and ultimately drives disease symptoms, characterized by a lack of coordination and muscle control during voluntary movements.
SGT-212 aims to deliver a healthy version of a frataxin-encoding gene to the body’s cells.
The therapy is administered to patients through a dual route: It’s given directly into a specific brain region to help address neurological issues, and it’s also administered intravenously, or into the bloodstream, with the aim of improving heart health.
SGT-212 now being tested in adults in small trial
The gene therapy candidate is now being tested in adults in a Phase 1 clinical trial dubbed FALCON (NCT07180355).
FALCON is expected to enroll about 10 people, ages 18 to 40, with a confirmed diagnosis of Friedreich’s ataxia. To be eligible, patients must have started to experience symptoms before age 25, and must currently have a score of 1 to 5 on the Friedreich’s Ataxia Rating Scale (FARS) Functional Disability Staging Score. Patients taking the approved therapy Skyclarys (omaveloxolone) must be on a stable dose for at least three months before enrolling, and must expect to continue taking the drug at that dose throughout the study.
All participants in FALCON will be treated with SGT-21. The trial’s main goal is to assess safety outcomes in the first year after the gene therapy is administered.
The trial, which is being run at two sites in Ohio and Pennsylvania, is now screening participants, according to Solid. All participants will be enrolled for approximately five years.
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.
In announcing the new designation, Solid noted that, as of now, there are “no treatments that provide a cure or halt disease progression” in FA. The developer said this new FDA status will help advance its experimental therapy.
“These designations are designed to help accelerate time to market and enhance engagement with the FDA,” Hanrahan said. “We look forward to continued collaboration with regulators to bring this therapy to patients as quickly as possible.”
About the Author
