What is the study drug (belumosudil)?

The study drug is a tablet designed to target the ROCK2 pathway. Pathways are how cells in the body communicate with each other to help our bodies function. When ROCK2 is overactivated, it may result in inflammation of the skin and damage to internal organs. One goal of the study is to see if targeting ROCK2 can help with the symptoms of scleroderma.

Belumosudil has been approved by the FDA for the treatment of adult and pediatric patients 12 years and older with chronic graft-versus-host disease (chronic GVHD) after failure of at least two prior lines of systemic therapy. However, it is not approved for scleroderma or any other condition. Belumosudil is currently being studied for investigational use in people with scleroderma.

What is the investigational use of a drug?

The investigational use of a drug means that a drug is being used in a new way, but that new use has not been approved by regulatory authorities, such as the United States Food and Drug Administration (FDA) or the European Medicines Agency (EMA). Investigational uses are uses that are still being researched to determine if the drug works and is safe for that use. 

Have other people used belumosudil before?

Yes. Previous clinical studies have researched the safety and effectiveness of the study drug for other conditions that may also be caused by a malfunctioning ROCK2 pathway. Findings from these studies suggest that targeting ROCK2 may reduce inflammation in conditions like scleroderma.