Solid Biosciences宣布FDA授予SGT-212罕见儿科疾病称号，SGT-212是其治疗弗里德赖希共济失调的研究性基因疗法

Solid Biosciences Inc. (NASDAQ:SLDB) (the "Company" or "Solid"), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that it received Rare Pediatric Disease designation from the U.S. Food and Drug Administration (FDA) for SGT-212, the Company's investigational gene therapy for Friedreich's ataxia (FA). SGT-212 will deliver the full-length frataxin gene via dual routes of administration, utilizing both direct intradentate nucleus (IDN) and intravenous (IV) infusions, and was designed to promote restoration of therapeutic levels of the frataxin protein to address neurologic, cardiac and systemic clinical manifestations of FA.

Rare Pediatric Disease designation is granted by the FDA for serious and life-threatening diseases that primarily affect individuals under 18 years of age. The designation provides the Company with the potential to receive a pediatric priority review voucher (PRV), which can expedite the review for future Biologic License Applications. The PRV may be redeemed to receive priority review for another marketing application or may be sold or transferred.

Jessie Hanrahan, Ph.D., Chief Regulatory & Preclinical Operations Officer of Solid Biosciences, said, "Receiving Pediatric Rare Disease designation marks another significant milestone for our Friedreich's ataxia program, SGT-212. 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. 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."