An emerging biotech startup out of Rosalind Franklin University's biomedical incubator, Helix 51, said Thursday it received a key designation from the U.S. Food and Drug Administration for BLR-200, a treatment of systemic sclerosis, also known as scleroderma.
BLR Bio, a company that is developing treatments for cancer and a broad spectrum of fibrotic diseases, received an Orphan Drug Designation for its scleroderma treatment. The effort targets a key scar-forming cell to treat the rare rheumatic disease.
Scleroderma affects connective tissue and the vascular system and causes fibrosis in the skin or in internal organs, resulting in disfigurement and disabilities. The most common symptoms are the loss of mobility and function, pain, and fatigue. There is no cure to scleroderma, and the cause is unknown.
The FDA's ODD program provides incentives to sponsor organizations for the development of innovative treatments for rare diseases that affect fewer than 200,000 people in the United States.
"The FDA's granting of Orphan Drug Designation to BLR-200 highlights the urgent need for new and innovative therapeutic options for patients afflicted with the disease," said BLR Bio CEO Bruce Riser in a statement.
