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.
Select a button below to view the list of FDA events for that drug.
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:
- May 7, 2026
- Indication:
- For Duchenne Muscular Dystrophy Gene Therapy
Announcement
Solid Biosciences announced that the first participant has been dosed in IMPACT DUCHENNE, the Company's multi-country, placebo-controlled, randomized, double-blind, Phase 3 clinical trial investigating SGT-003 for the treatment of Duchenne muscular dystrophy (Duchenne).
AI Summary
Solid Biosciences announced that the first participant has been dosed in IMPACT DUCHENNE, its multi-country, placebo-controlled, randomized, double-blind Phase 3 trial evaluating SGT-003 for Duchenne muscular dystrophy. The initial dose was given in Australia at The Children’s Hospital at Westmead. The trial is designed to test safety and efficacy in a well-controlled, blinded setting and will contribute key evidence about SGT-003’s potential benefits for people with Duchenne.
IMPACT DUCHENNE is part of Solid’s integrated development program aimed at supporting registration and global regulatory authorizations for SGT-003. That program also includes the Phase 1/2 INSPIRE trial, which has dosed 46 participants using a steroid-only prophylactic immunomodulation regimen; SGT-003 was generally well tolerated as of the May 4, 2026 cutoff. The Phase 3 launch reflects the company’s push to generate rigorous data to potentially speed access for patients with this serious, progressive disease.
Read Announcement- Drug:
- SGT-003
- Announced Date:
- March 11, 2026
- Indication:
- For Duchenne Muscular Dystrophy Gene Therapy
Announcement
Solid Biosciences reported updated positive interim data from the ongoing Phase 1/2 INSPIRE DUCHENNE clinical trial of SGT-003.
AI Summary
Solid Biosciences reported positive interim data from the Phase 1/2 INSPIRE DUCHENNE trial of SGT-003, with 40 participants dosed as of March 11, 2026. At a single intravenous dose of 1E14 vg/kg, SGT-003 produced robust microdystrophin expression and consistent evidence of dystrophin-associated protein complex (DAPC) restoration. The company observed improvements in muscle integrity through a broad biomarker panel at Day 90 and Day 360, suggesting healthier, more stable muscle fibers and less ongoing damage. Cardiac function, measured by LVEF, showed stabilization or improvement after treatment.
SGT-003 was generally well tolerated in the 40 participants and is given with a lower-burden, steroid-only prophylactic immunomodulation regimen. The open-label trial is ongoing at 15 sites in the US, Canada, Italy and the UK. Solid plans additional meetings with the FDA in H1 2026 to discuss a potential accelerated approval pathway and expects an update mid-2026.
Read Announcement- Drug:
- SGT-003
- Announced Date:
- February 9, 2026
- Indication:
- For Duchenne Muscular Dystrophy Gene Therapy
Announcement
Solid Biosciences announced a positive regulatory update from its recent Type C meeting with the U.S. Food and Drug Administration (FDA) that supports the continued advancement of SGT-003 as a potential treatment for Duchenne muscular dystrophy.
AI Summary
Solid Biosciences announced a positive regulatory update from a recent Type C meeting with the U.S. Food and Drug Administration that supports continued advancement of SGT-003 as a potential treatment for Duchenne muscular dystrophy. The FDA and company are aligned on a Phase 3 randomized, double-blind, placebo-controlled trial design (IMPACT DUCHENNE) to evaluate a single dose in ambulatory participants aged 7 to under 12. The company expects first participant dosing in Q1 2026 and describes the multinational study as intended to support potential regulatory authorizations.
Solid Biosciences also plans additional meetings with the FDA in the first half of 2026 to discuss a potential accelerated approval pathway for SGT-003. In earlier clinical work, SGT-003 has been generally well tolerated, with 36 participants dosed in the Phase 1/2 INSPIRE DUCHENNE trial as of February 9, 2026. The company said the FDA feedback helps move the program into Phase 3 with clearer regulatory alignment.
Read Announcement- Drug:
- SGT-003
- Announced Date:
- October 1, 2025
- Indication:
- For Duchenne Muscular Dystrophy Gene Therapy
Announcement
Solid Biosciences Inc. will present data from its neuromuscular and cardiac programs at the World Muscle Society (WMS) 2025 Annual International Congress, October 7-11, 2025, in Vienna, Austria, and at the European Society of Gene & Cell Therapy (ESGCT) 2025 Annual Congress, October 7-12, 2025, in Seville, Spain.
AI Summary
Solid Biosciences Inc. will present data from its neuromuscular and cardiac programs at the World Muscle Society 2025 Annual International Congress in Vienna, Austria (October 7–11), and at the European Society of Gene & Cell Therapy 2025 Annual Congress in Seville, Spain (October 7–12).
At WMS, the company will share clinical trial updates on its next-generation microdystrophin therapy SGT-003 for Duchenne muscular dystrophy, plus a poster on biomarkers of muscle integrity. At ESGCT, Solid Biosciences will present four posters on gene therapy advances: SGT-003 using the AAV-SLB101 capsid; cardiac gene transfer amid neutralizing antibodies; CASQ2 overexpression to correct arrhythmias; and an EV-AAV delivery method that shields transgenes. These presentations highlight the potential of its rationally designed capsid and its innovative pipeline. After the meetings conclude, all materials will be available on the Solid Biosciences website.
Read Announcement- 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:
- March 6, 2026
- Indication:
- For Treat Both Neurologic and Cardiac Manifestations of Friedreich's Ataxia
Announcement
Solid Biosciences Inc announced presentations on SGT-003, its next-generation investigational gene therapy for Duchenne muscular dystrophy, and SGT-212, its dual-route investigational gene therapy for Friedreich's ataxia (FA), at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place in Orlando, Florida, March 8-11, 2026.
AI Summary
Solid Biosciences announced it will present data on SGT-003, its next-generation microdystrophin gene therapy for Duchenne muscular dystrophy, and SGT-212, a dual-route FXN gene therapy for Friedreich’s ataxia, at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference in Orlando, March 8–11, 2026. An oral presentation, “Update on the INSPIRE DUCHENNE Phase 1/2 Study of SGT-003,” is scheduled for March 11 at 1:00 p.m. ET in Florida 4 and will be presented by Dr. Aravindhan Veerapandiyan.
Solid will also exhibit multiple posters on March 10 covering its next-generation capsid AAV-SLB101 (PK/PD effects), SGT-003 safety and liver toxicity, DAPC stabilization and muscle integrity, high cardiac tropism with AAV-SLB101, and the efficacy and safety of SGT-212 for FA. Presenters include Matthew Harmelink, Patrick Gonzalez, and Jun Lee. The company will discuss the INSPIRE trial (first-in-human Phase 1/2 for pediatric Duchenne) and FALCON (Phase 1b for adults with FA and cardiac hypertrophy).
Read Announcement- Drug:
- SGT-212
- Announced Date:
- January 12, 2026
- Indication:
- For Treat Both Neurologic and Cardiac Manifestations of Friedreich's Ataxia
Announcement
Solid Biosciences Inc. announced that the first participant has been dosed in FALCON, the Company's Phase 1b, first-in-human clinical trial evaluating SGT-212, its investigational gene therapy for the treatment of Friedreich's ataxia (FA).
AI Summary
Solid Biosciences announced that the first participant has been dosed in FALCON, its Phase 1b first‑in‑human trial of SGT‑212, an investigational AAV gene therapy for Friedreich’s ataxia (FA). SGT‑212 uses a dual‑route approach: a precise, stereotactic, MRI‑guided infusion into the cerebellar dentate nuclei followed by an intravenous infusion. The strategy aims to raise frataxin levels in the brain, heart and other tissues to address the neurologic, cardiac and systemic problems of FA.
Intra‑procedural MRI confirmed dentate nuclei targeting and coverage that exceeded the company’s expectations, bolstering confidence in the delivery method. FALCON is an open‑label, multicenter Phase 1b study in U.S. participants aged 18–40 with FA and cardiac hypertrophy. Solid said it will share preliminary safety observations in the coming months and expects an initial data update in the second half of 2026, subject to enrollment.
Read Announcement- Drug:
- SGT-212
- Announced Date:
- December 1, 2025
- Indication:
- For Treat Both Neurologic and Cardiac Manifestations of Friedreich's Ataxia
Announcement
Solid Biosciences Inc. 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).
AI Summary
Solid Biosciences announced it received Rare Pediatric Disease designation from the U.S. Food and Drug Administration for SGT-212, its investigational gene therapy for Friedreich’s ataxia (FA). The designation is given for serious, life‑threatening conditions that primarily affect children and could make the company eligible for a pediatric priority review voucher (PRV). A PRV can be used to speed review of a future biologics application or may be sold or transferred.
SGT-212 is an AAV‑based gene replacement designed to deliver full‑length frataxin via a dual‑route approach: a direct intradentate nucleus (IDN) infusion using an MRI‑guided stereotactic device, followed by an intravenous (IV) infusion to reach the heart. This strategy aims to restore frataxin levels in the cerebellar dentate nuclei and cardiomyocytes to address neurologic, cardiac and systemic symptoms of FA; targeted delivery to the dentate nuclei will be confirmed in real time with gadolinium. The FALCON Phase 1b first‑in‑human trial is currently screening participants.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 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 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 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 23, 2025
- Target Action Date:
- Q4 2025
- Estimated Target Date Range:
- October 1, 2025 - December 31, 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 is developing a precision gene therapy for catecholaminergic polymorphic ventricular tachycardia (CPVT), a rare inherited heart disorder that causes fast, irregular heartbeats leading to fainting or sudden death.
Their investigational therapy, SGT-501, uses a harmless viral vector to deliver a healthy copy of the CASQ2 gene into heart muscle cells. By restoring proper calcium handling and stabilizing the ryanodine receptor, SGT-501 aims to correct the root cause of dangerous arrhythmias.
Solid Biosciences expects to start a Phase 1b clinical trial of SGT-501 in the fourth quarter of 2025. This first-in-human, open-label, multicenter study will assess the treatment’s safety, tolerability and early signs of effectiveness in people with CPVT, paving the way for potential new options.
Read Announcement- Drug:
- SGT-501
- Announced Date:
- July 23, 2025
- Indication:
- For the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT)
Announcement
Solid Biosciences Inc announced that it received Fast Track designation from the U.S. Food and Drug Administration (FDA) for SGT-501, the Company's novel, AAV-based investigational gene therapy for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT).
AI Summary
Solid Biosciences announced that SGT-501, its novel AAV-based investigational gene therapy for catecholaminergic polymorphic ventricular tachycardia (CPVT), received Fast Track designation from the U.S. Food and Drug Administration. This status is granted to therapies that treat serious, life-threatening conditions and show promise in addressing unmet medical needs.
SGT-501 works by delivering a full-length, codon-optimized copy of the human CASQ2 gene to heart muscle cells. By boosting calsequestrin protein levels, it aims to stabilize the ryanodine receptor (RYR2) and correct calcium regulation, helping to normalize heart rhythm and prevent dangerous arrhythmias in CPVT patients.
In addition to Fast Track, SGT-501 holds Orphan Drug and Rare Pediatric Disease designations and is on track to start its first human Phase 1b trial in the fourth quarter of 2025. If successful, it could become the first therapy targeting the root causes of CPVT.
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- 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
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