This section highlights FDA-related milestones and regulatory updates for drugs developed by Solid Biosciences (SLDB).
Over the past two years, Solid Biosciences has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
SGT-003, SGT-212, and SGT-501. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
SGT-003 - FDA Regulatory Timeline and Events
SGT-003 is a drug developed by Solid Biosciences for the following indication: For Duchenne Muscular Dystrophy Gene Therapy.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- SGT-003
- Announced Date:
- March 6, 2025
- Indication:
- For Duchenne Muscular Dystrophy Gene Therapy
Announcement
Solid Biosciences provided a business update.
AI Summary
Solid Biosciences provided a business update highlighting significant progress in its clinical programs. The company has shown early promising safety data and biomarker improvements in its Phase 1/2 INSPIRE DUCHENNE trial with SGT-003, a next-generation gene therapy candidate for Duchenne muscular dystrophy. In mid-2025, the company plans to request a meeting with the U.S. Food and Drug Administration to discuss potential accelerated approval pathways for SGT-003, aiming to quickly move the therapy through the clinic.
Additionally, Solid Biosciences shared a robust financial update. The company ended 2024 with $148.9 million in cash, cash equivalents, and investments. With the $200 million gross proceeds from a recent offering, the business expects a strong cash runway into the first half of 2027. These advancements support the company’s broader goal of developing precision genetic medicines for neuromuscular and cardiac diseases.
Read Announcement- Drug:
- SGT-003
- Announced Date:
- February 18, 2025
- Indication:
- For Duchenne Muscular Dystrophy Gene Therapy
Announcement
Solid Biosciences announced positive initial data from the Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation gene therapy product candidate intended for the treatment of Duchenne muscular dystrophy (Duchenne).
AI Summary
Solid Biosciences announced positive initial data from its Phase 1/2 INSPIRE DUCHENNE trial for SGT-003, a next-generation gene therapy candidate for Duchenne muscular dystrophy. In the first three participants, a 90‐day biopsy revealed an average microdystrophin expression of 110%, measured using multiple methods. These promising results were accompanied by significant improvements in key muscle health biomarkers, indicating enhanced muscle integrity.
The trial also showed early signals of potential cardiac benefit, with improvements noted in cardiac function in some participants. SGT-003 was well-tolerated among the six participants dosed so far, with no serious adverse events observed. This initial clinical data reinforces the potential of SGT-003 as a best-in-class gene therapy for treating Duchenne, offering hope for better management of the disease in young patients as further data is collected.
Read Announcement- Drug:
- SGT-003
- Announced Date:
- January 15, 2025
- Indication:
- For Duchenne Muscular Dystrophy Gene Therapy
Announcement
Solid Biosciences Inc. will provide a corporate update outlining the Company's expanded clinical-stage pipeline and 2025 objectives as it completes its transformation into a multi-program leader in the development of precision genetic medicines. Bo Cumbo, President and CEO, and Gabriel Brooks, M.D., Chief Medical Officer, will provide the update at the 43rd Annual J.P. Morgan Healthcare Conference on Wednesday, January 15, 2025, at 3:45 p.m. PT (6:45 p.m. ET).
AI Summary
Solid Biosciences Inc. will provide a corporate update at the 43rd Annual J.P. Morgan Healthcare Conference, focusing on its expanded clinical-stage pipeline and 2025 objectives as it transforms into a multi-program leader in precision genetic medicines. During the conference, President and CEO Bo Cumbo and Chief Medical Officer Dr. Gabriel Brooks will present at 3:45 p.m. PT (6:45 p.m. ET) on January 15, 2025. Their update will highlight the company’s progress in broadening its focus beyond genetic treatments for Duchenne muscular dystrophy to include innovative therapies for various neuromuscular and cardiac diseases. The presentation is expected to provide insights into new and improved gene therapy treatments, underlining the firm’s commitment to developing transformative medicines. This strategic update aims to outline how Solid Biosciences plans to meet critical patient needs and create further value for its shareholders.
Read Announcement
SGT-212 - FDA Regulatory Timeline and Events
SGT-212 is a drug developed by Solid Biosciences for the following indication: For Treat Both Neurologic and Cardiac Manifestations of Friedreich's Ataxia.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- SGT-212
- Announced Date:
- January 21, 2025
- Indication:
- For Treat Both Neurologic and Cardiac Manifestations of Friedreich's Ataxia
Announcement
Solid Biosciences Inc announced that it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for SGT-212, the Company's, AAV-based gene therapy candidate for the treatment of Friedreich's ataxia (FA).
AI Summary
Solid Biosciences Inc. recently received Fast Track designation from the U.S. Food and Drug Administration (FDA) for its gene therapy candidate SGT-212, which targets Friedreich’s ataxia (FA). This designation is granted for treatments that address serious or life-threatening conditions and meet an unmet medical need, and it allows for more frequent interactions with the FDA to help speed up development and review processes.
SGT-212 is an AAV-based gene therapy designed to deliver the full-length frataxin gene using a dual route of administration. Specifically, it involves an injection into the brain's dentate nucleus along with an intravenous infusion to restore therapeutic levels of frataxin. By addressing the neurologic, cardiac, and systemic problems associated with FA, this innovative treatment could offer new hope for patients suffering from this devastating disease.
Read Announcement- Drug:
- SGT-212
- Announced Date:
- January 21, 2025
- Indication:
- For Treat Both Neurologic and Cardiac Manifestations of Friedreich's Ataxia
Announcement
Solid Biosciences Inc announced that it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for SGT-212, the Company's, AAV-based gene therapy candidate for the treatment of Friedreich's ataxia (FA).
AI Summary
Solid Biosciences Inc. announced that it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for its gene therapy candidate, SGT-212, which is aimed at treating Friedreich’s ataxia (FA). SGT-212 is an innovative AAV-based therapy designed to deliver the full-length frataxin gene. The treatment uses a dual route delivery system, combining intradentate nucleus (IDN) and intravenous (IV) infusions, to restore frataxin levels in both the brain and heart. This method is intended to address the neurological and cardiac symptoms associated with FA.
The Fast Track designation recognizes the urgent need for new treatments for serious and life-threatening conditions like FA and may help accelerate the development and review process for SGT-212. Solid Biosciences hopes that this expedited pathway will bring meaningful progress and hope to patients suffering from this debilitating disease.
Read Announcement- Drug:
- SGT-212
- Announced Date:
- January 7, 2025
- Indication:
- For Treat Both Neurologic and Cardiac Manifestations of Friedreich's Ataxia
Announcement
Solid Biosciences Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for SGT-212 for the treatment of Friedreich's ataxia (FA), a degenerative disease caused by insufficient levels of the frataxin protein.
AI Summary
Solid Biosciences Inc. announced that the FDA has approved its Investigational New Drug (IND) application for SGT-212, a gene therapy candidate aimed at treating Friedreich's ataxia (FA). FA is a degenerative disease caused by low levels of the frataxin protein, leading to severe neurological and cardiac issues. SGT-212 is unique in that it delivers the full-length frataxin gene through both systemic intravenous infusion and direct infusion into the cerebellum. This dual method is designed to target the central nervous system and heart, the two areas most affected by FA. The clearance by the FDA marks an important milestone, supporting further clinical development. Solid Biosciences plans to start its Phase 1b clinical trial in the second half of 2025 to assess the safety and effectiveness of this innovative therapy, providing hope for improved treatment of FA symptoms.
Read Announcement
SGT-501 - FDA Regulatory Timeline and Events
SGT-501 is a drug developed by Solid Biosciences for the following indication: For the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- SGT-501
- Announced Date:
- July 8, 2025
- Indication:
- For the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT)
Announcement
Solid expects to initiate Phase 1b clinical trial of SGT-501 in the fourth quarter of 2025 -
AI Summary
Solid Biosciences announced plans to begin a Phase 1b clinical trial for its novel gene therapy, SGT-501, in the fourth quarter of 2025. The therapy aims to treat catecholaminergic polymorphic ventricular tachycardia (CPVT), a rare and life-threatening genetic heart disorder characterized by dangerous arrhythmias. SGT-501 uses an AAV-based approach to deliver a functioning copy of the CASQ2 gene into heart cells, which may improve calcium handling and stabilize heart rhythms.
The upcoming trial will assess the safety, tolerability, and effectiveness of SGT-501 in patients who currently lack approved treatment options. This effort marks Solid Biosciences’ first venture into a cardiac indication and addresses an urgent unmet need, potentially offering patients a durable treatment solution that could reduce the risk of lethal arrhythmias and improve their quality of life.
Read Announcement- Drug:
- SGT-501
- Announced Date:
- July 8, 2025
- Indication:
- For the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT)
Announcement
Solid Biosciences Inc. announced approval of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) and clinical trial application (CTA) by Health Canada for SGT-501, a novel gene therapy approach for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), a highly malignant, arrhythmogenic channelopathy caused by genetic mutations that impact the ryanodine receptor (RYR2) in cardiac muscle.
AI Summary
Solid Biosciences Inc. announced that the U.S. FDA has approved its Investigational New Drug (IND) application and Health Canada has approved the clinical trial application (CTA) for SGT-501. This novel gene therapy targets catecholaminergic polymorphic ventricular tachycardia (CPVT), a severe cardiac condition caused by mutations affecting the ryanodine receptor (RYR2). CPVT leads to dangerous irregular heart rhythms triggered by stress or exercise and currently has no approved treatments. The therapy aims to correct the underlying calcium regulation problems in heart muscle cells by delivering a functional copy of the gene involved, potentially stopping the life-threatening arrhythmias. Solid Biosciences plans to start a Phase 1b clinical trial in the fourth quarter of 2025 to assess the safety, tolerability, and effectiveness of SGT-501 for patients with this critical heart condition.
Read Announcement
