The Food and Drug Administration (FDA) has granted Orphan Drug designation to TRE-515 for the treatment of demyelinating optic neuritis.

Optic neuritis (ON) is a rare autoimmune disease characterized by the inflammation of the optic nerve. The condition can lead to rapid loss of vision in one or both eyes. TRE-515 is a potential first-in-class, orally administered, small molecule inhibitor of deoxycytidine kinase (dCK), the rate-limiting enzyme in the nucleoside salvage pathway. 

“Inflammation damaging the protective myelin coating around the optic nerve causes ON, leading to pain and vision loss,” said Dr Peter Clark, member of the Trethera Scientific Advisory Board. “Targeting dCK with TRE-515 could limit this inflammation.”

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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.

“TRE-515 could potentially significantly benefit ON patients beyond the available therapeutic options, especially those taking long-term steroids,” said Trethera Scientific Advisory Board member Dr Larry Steinman.


Trethera announces FDA Orphan Drug designation granted to TRE-515 for the treatment of demyelinating optic neuritis. News release. Trethera Corporation. Accessed August 11, 2022.

This article originally appeared on MPR