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# Orphan Status for Syntimmune Pemphigus Therapy
- URL: https://www.fdaweb.com/orphan-status-for-syntimmune-pemphigus-therapy/
- Published: 2018-09-12T12:00:00.000Z
- Updated: 2026-09-15T00:31:23.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5142339

FDA has granted Syntimmune an orphan drug designation for SYNT001 for treating pemphigus. The designation is based on positive preliminary results from a Phase 1b proof-of-concept trial in pemphigus patients, a rare and severe autoimmune disease that leads to painful blistering. Data showed that SYNT001 appeared to be well-tolerated and induced a rapid reduction in Immunoglobulin G (IgG) and circulating immune complex levels, as well as clinical improvement as measured by the Pemphigus Disease Area Index score, with clinical effect persisting beyond the treatment period, the company says.