FDA Grants Orphan Drug Designation for Treatment of Scleroderma Developed by Helix 51's BLR Bio

The U.S. Food and Drug Administration (FDA) granted an Orphan Drug Designation for an investigational therapy under development by Helix 51 company BLR Bio for the treatment of systemic sclerosis (SSc), also known as scleroderma.

Orphan Drug Designation is granted by the FDA to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the U.S. The status provides benefits to drug developers, including assistance in the development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of post-approval marketing exclusivity.

According to the National Scleroderma Foundation, scleroderma is a rare rheumatic disease that affects connective tissue and the vascular system, producing excessive collagen that causes fibrosis in the skin (localized) or in internal organs (systemic sclerosis). The result can be disfigurement and disability. Patients commonly experience loss of mobility and function, pain and fatigue. 

The cause of scleroderma is unknown and there is no cure. It has the highest mortality rate among rheumatic diseases, with a 2.5-fold higher risk of death than the general population. 

The investigational therapy, BLR-200, is different from other therapies now in development for the treatment of systemic sclerosis.

“Our therapy addresses multiple disease-supporting elements or redundancies in the pathways to initiation and progression, and targets a key scar-forming cell population of myofibroblasts,” said Bruce Riser, PhD, CEO of BLR Bio. “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.”

BLR Bio was awarded a $780,000 grant in collaboration with expert investigators at the University of Saskatchewan in Saskatoon, Canada, from the Canadian Institutes of Health Research in 2022 to further study BLR-200 in scleroderma. This research is ongoing and has demonstrated promising in vivo results.

Executive Vice President for Research Ronald Kaplan, PhD, said the orphan designation represents an important milestone in the development of BLR Bio’s drug candidate.

“BLR-200 has the potential to establish first-in-class clinical benefits by addressing multiple elements driving scleroderma and treating multiple organs negatively impacted by the disease,” Dr. Kaplan said.

To learn more about BLR Bio, visit blrbio.com.