Benlysta granted Orphan Drug Designation by US FDA for the potential treatment of systemic sclerosis

GSK plc announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Benlysta (belimumab), a B-cell inhibiting monoclonal antibody, for the potential treatment of systemic sclerosis. GSK plans to initiate a phase II/III trial of belimumab for systemic sclerosis associated interstitial lung disease (SSc-ILD) in the first half of 2023.

Systemic sclerosis (SSc) is a rare autoimmune disease that causes atypical growth of connective tissues and can affect the musculoskeletal system, heart, lungs, kidneys, skin, and other organs. Interstitial lung disease (ILD) is the leading cause of death in SSc, affecting as many as half of people living with the disease.

With limited treatment options available for SSc-ILD, this Orphan Drug Designation reflects the need for further research and the potential for belimumab to address a critical need for people living with this debilitating condition. GSK continues to follow the science to explore how belimumab may be able to address an unmet need in B-cell-driven autoimmune diseases.

The US FDA’s ODD is a special status granted to support the development and evaluation of potential new medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US.