Solid Biosciences. FDA Fast Track Designation granted on July 23, 2025, for (SGT-501) a first-in-class gene therapy for Catecholaminergic polymorphic ventricular tachycardia (CPVT).
Solid Biosciences.
FDA Fast Track Designation granted on July 23, 2025, for (SGT-501) a first-in-class gene therapy for Catecholaminergic polymorphic ventricular tachycardia (CPVT).
Key Notes:
1. Condition Overview:
Catecholaminergic polymorphic ventricular tachycardia (CPVT) is a rare inherited heart rhythm disorder that increases the risk of life-threatening ventricular tachycardia (VT) and cardiac arrest.
2. Lack of Current Treatments:
There are currently no FDA-approved pharmaceutical treatments for CPVT. Management focuses on reducing VT risk using indirect interventions.
3. New Therapy – SGT-501:
Solid Biosciences developed SGT-501, a gene therapy that delivers a healthy version of the human cardiac calsequestrin gene directly to heart muscle cells. This targets the genetic cause of CPVT.
4. FDA Support:
• SGT-501 previously received IND (Investigational New Drug) clearance.
• In July 2025, it was granted FDA Fast Track Designation, highlighting the urgent need for therapies in this area.
5. Clinical Trial Plan:
A first-in-human clinical trial of SGT-501 is scheduled to begin in late 2025.
6. Company Perspective:
Solid Biosciences emphasized the therapy’s strong preclinical and manufacturing data and sees SGT-501 as a potentially durable and transformative solution for CPVT.
7. Regulatory Advantages:
Fast Track designation allows:
• More frequent interaction with the FDA
• Faster feedback and document review
• Potential for accelerated approval if early results are promising