The Food and Drug Administration (FDA) has granted Orphan Drug designation to cudetaxestat 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. 

Autotaxin, a key enzyme important for generating the signaling lipid lysophosphatidic acid, is highly expressed in various fibrotic diseases and develops in response to epithelial cell/tissue damage. In early studies, cudetaxestat, a noncompetitive, reversible inhibitor of autotaxin, was found to be well tolerated and demonstrated direct antifibrotic activity. 

“There are limited options and high medical need for patients suffering from systemic sclerosis,” said Daven Mody, PharmD, MBA, vice president of regulatory affairs with Blade. “We look forward to continuing our clinical development efforts for cudetaxestat in lung fibrosis and potentially exploring other areas of high medical need such as [systemic sclerosis].”

The FDA’s Orphan Drug designation is granted to medicines intended to treat or prevent rare diseases or disorders that affect fewer than 200,000 individuals. Cudetaxestat was previously granted Orphan Drug designation for idiopathic pulmonary fibrosis.


Blade Therapeutics announces FDA Orphan Drug designation granted to cudetaxestat for treatment of systemic sclerosis. News release. Blade Therapeutics, Inc. Accessed October 14, 2021.