FDA Grants Breakthrough Therapy Status to Veligrotug for Thyroid Eye Disease

Viridian Therapeutics, Inc., a biopharmaceutical company focused on innovative treatments for serious and rare diseases, has achieved a significant milestone with its lead drug candidate, veligrotug. The United States Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to veligrotug, an anti-insulin-like growth factor-1 receptor (IGF-1R) drug aimed at treating Thyroid Eye Disease (TED). This designation is awarded to promising drugs that show potential for substantial improvement over existing therapies for serious conditions.

Veligrotug's designation was based on its consistent and robust improvement in treating diplopia, a common symptom of chronic TED, and its rapid onset of response in reducing proptosis. Diplopia, or double vision, is a challenging symptom for many TED patients, and the therapy showed notable efficacy in resolving it. The drug's rapid action, with effects observable within three weeks after a single infusion, highlights its potential as a significant advancement in TED treatment.

The drug candidate proved successful in the pivotal THRIVE and THRIVE-2 clinical trials, which are the most comprehensive studies conducted to date for TED. Veligrotug met all of its primary and secondary endpoints, demonstrating both effectiveness and safety in patients with active and chronic forms of the disease. Notably, THRIVE-2 was the first global phase 3 trial to demonstrate a statistically significant response in reducing diplopia among chronic TED patients, marking a first in the field.

Veligrotug's intravenous (IV) formulation targets the IGF-1R, a validated and commercially significant target for TED therapies. The drug is designed to improve the treatment experience for patients, offering a shorter infusion time and fewer sessions compared to existing IGF-1R inhibitors. This could represent a meaningful improvement for patients, given the burdensome nature of current treatment regimens.

Viridian is now on track to submit a Biologics License Application (BLA) for veligrotug by the second half of 2025, with hopes of launching the drug in the U.S. market in 2026, pending approval. The Breakthrough Therapy Designation supports veligrotug's eligibility for Priority Review, potentially expediting its path to market.

Viridian, headquartered in Waltham, Massachusetts, is dedicated to developing best-in-class medicines. The company leverages its expertise in antibody discovery and protein engineering to create innovative therapies for validated targets in commercially viable disease areas. Beyond veligrotug, Viridian is advancing VRDN-003, a potential subcutaneous IGF-1R antibody therapy for TED, currently in phase 3 trials. The company is also exploring treatments for other autoimmune diseases through its novel portfolio of neonatal Fc receptor inhibitors.

With veligrotug, Viridian is poised to make a significant impact in the TED treatment landscape. The drug's demonstrated efficacy and safety, coupled with the innovative approach to dosing and administration, position it as a potential new standard of care for TED patients. As the company progresses toward regulatory submissions and potential commercialization, the Breakthrough Therapy Designation marks an encouraging step forward in addressing unmet needs in the field of thyroid eye disease.