The Food and Drug Administration (FDA) has granted Orphan Drug designation to TRE-515 for the treatment of acute disseminated encephalomyelitis (ADEM).

ADEM is a rare neurological, autoimmune disease characterized by inflammation of the central nervous system causing damage to myelin. Patients with ADEM lose nerve function, which can ultimately lead to paralysis and death in some cases.

TRE-515 is a potential first-in-class, orally bioavailable, small molecule inhibitor of deoxycytidine kinase, which is the rate-limiting enzyme in the nucleoside salvage pathway. Based on its mechanism of action, it is believed that certain autoimmune diseases will respond to TRE-515 treatment.

“No medications have been specifically approved by the FDA to treat [ADEM],” said Dr Peter Clark, a specialist member of the Trethera Scientific Advisory Board. “Holding [Orphan Drug Status] at this early preclinical stage is a mark of distinction and external validation.”

Trethera is also investigating TRE-515 as a potential treatment for solid tumors as the nucleoside salvage pathway may play a role in rapid cell proliferation of cancer cells.


Trethera announces FDA Orphan Drug designation granted to TRE-515 in the treatment of acute disseminated encephalomyelitis. News release. Trethera Corporation. Accessed August 30, 2022.