Viridian Therapeutics Receives FDA Breakthrough Therapy Designation
Viridian Therapeutics Receives FDA Breakthrough Therapy Designation for Veligrotug for the Treatment of Thyroid Eye Disease (TED)
WALTHAM, Mass. -- Viridian Therapeutics, Inc., a biopharmaceutical company focused on discovering, developing, and commercializing potential best-in-class medicines for serious and rare diseases, today announced that the United States Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to veligrotug (“veli”), the company’s lead anti-insulin-like growth factor-1 receptor (IGF-1R) drug candidate for the treatment of TED.
Breakthrough Therapy Designation is reserved for drug candidates intended to treat serious or life-threatening conditions that address an unmet need where clinical evidence has indicated they may demonstrate substantial improvement on a clinically significant endpoint over existing therapies.
“The FDA’s decision to grant veli Breakthrough Therapy Designation underscores that veli may offer substantial improvement over existing therapies, and we believe it reinforces veli’s potential to be a differentiated, treatment-of-choice for patients living with TED,” said Steve Mahoney, Viridian’s President and CEO. “Veli is the only therapy that has demonstrated statistically significant and clinically meaningful improvement and resolution of diplopia in both active and chronic TED. Veli also showed a rapid onset of treatment effect, including an improvement in proptosis response in as few as three weeks after just one infusion. We believe veli will be an important potential new treatment option for patients living with TED, including whose disease is unaddressed by existing therapies. We continue to execute on our planned BLA submission in the second half of 2025 and are excited that this designation supports eligibility for Priority Review as we prepare for our planned U.S. commercial launch in 2026.”
THRIVE and THRIVE-2, phase 3 clinical trials in active and chronic TED respectively, comprise the largest pivotal program to date in TED. In both clinical trials, veligrotug met all of its primary and secondary endpoints and was generally well-tolerated. THRIVE-2 was the first global phase 3 clinical trial to demonstrate a statistically significant diplopia response and resolution in chronic TED patients.
Veligrotug is an intravenously (IV) delivered, anti-insulin-like growth factor-1 receptor (IGF-1R) antibody in phase 3 development for thyroid eye disease, with the potential to be the IV treatment-of-choice for active and chronic TED patients. IGF-1R is a clinically and commercially validated target for thyroid eye disease (TED) with U.S. revenues of approximately $2 billion in 2024. Veligrotug has the potential to improve patient experience with a differentiated dosing regimen that features a shorter infusion time and fewer infusions compared to the currently approved and marketed IGF-1R inhibitor.
