Novel Treatment for Pemphigus Granted Orphan Drug Designation

Studies suggest that SYNT001 accomplishes its effects on IgG without destroying immune cells or impacting other types of immunoglobulin
Studies suggest that SYNT001 accomplishes its effects on IgG without destroying immune cells or impacting other types of immunoglobulin

The Food and Drug Administration (FDA) has granted Orphan Drug designation to SYNT001 (Syntimmune) for the treatment of pemphigus, a rare and severe autoimmune disease that causes painful blistering. 

SYNT001 is an investigational humanized IgG4 monoclonal antibody optimized to inhibit FcRn binding to IgG at both neutral and acidic pH. Preliminary results from a Phase 1b proof-of-concept study showed that SYNT001 was associated with clinical improvement (as measured by Pemphigus Disease Area Index score) and induced a rapid reduction in lgG and circulating immune complex levels; these effects persisted beyond the treatment period.  

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"We believe there is significant potential for SYNT001 in pemphigus and other autoimmune diseases and look forward to providing clinical data," said Jean-Paul Kress, MD, President and CEO of Syntimmune. 

For more information visit Syntimmune.com.