Belimumab Granted Orphan Drug Status for Systemic Sclerosis

systemic sclerosis systemic lupus erythematosus pulmonary arterial hypertension
systemic sclerosis systemic lupus erythematosus pulmonary arterial hypertension
Belimumab is a B-lymphocyte stimulator-specific inhibitor.

The Food and Drug Administration (FDA) has granted Orphan Drug designation to belimumab for the treatment of systemic sclerosis.

Systemic sclerosis is a rare, chronic autoimmune disorder characterized by degenerative changes and thickening of the skin and other organs from excessive collagen deposition. Elevated B-lymphocyte stimulator (BLyS) levels and autoreactive B cells have been reported to play a role in the pathogenesis of systemic sclerosis.

By binding to soluble BLyS, belimumab inhibits the survival of B cells, including autoreactive B cells, and reduces the differentiation of B cells into immunoglobulin-producing plasma cells. A phase 2/3 trial evaluating belimumab for systemic sclerosis associated interstitial lung disease is expected to begin in the first half of 2023.

Belimumab is currently marketed under the brand name Benlysta for the treatment of active systemic lupus erythematosus or active lupus nephritis in patients 5 years of age and older who are receiving standard therapy.


Benlysta granted Orphan Drug designation by US FDA for the potential treatment of systemic sclerosis. News release. GSK. Accessed February 1, 2023.