New gene therapy for rare heart condition gains FDA’s fast track designation
Solid Biosciences, a Massachusetts-based pharmaceutical company, has received the FDA’s fast track designation for a first-in-class gene therapy for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT).
CPVT is a rare heart rhythm disorder typically passed on from one or both of a person’s patients. It is associated with a heightened risk of or ventricular tachycardia (VT) and cardiac arrest. There are currently no pharmaceutical treatments for CPVT, though patients with the condition are often given specific treatments to combat the risk of VT.
The gene therapy in question, SGT-501, is designed to deliver a copy of the human cardiac calsequestrin gene to a patient’s heart muscle cells, normalizing symptoms brought on by CPVT. It previously received the investigational new drug (IND) clearance from the FDA. A new first-in-human clinical trial is now scheduled to begin in late 2025.
“FDA IND clearance and fast track designation provide important scientific validation of SGT-501 and recognition of the continuing and severe unmet needs posed by CPVT,” Jessie Hanrahan, PhD, chief regulatory and preclinical operations officer of Solid Biosciences, said in a statement. “We believe SGT-501’s robust preclinical and CMC data package supports the potential for a durable and transformational treatment for a disease that has no FDA-approved therapies. SGT-501 is our third IND clearance in the past two years, demonstrating Solid’s deep regulatory expertise in the genetic medicines space.”
The FDA’s fast track designation is intended to help speed up the approval process for companies developing treatments for life-threatening conditions, especially those that fill an unmet need. Drugs receiving this designation are eligible for more frequent meetings with the FDA, more written communication with the agency and accelerated approvals once certain criteria are met.
Michael has more than 19 years of experience as a professional writer and editor. He has written at length about cardiology, radiology, artificial intelligence and other key healthcare topics.