On September 12, 2018, the U.S. Food and Drug Administration (FDA) granted an orphan drug designation to Syntimmune Inc.’s SYNT001 for the treatment of pemphigus.
“This is an important milestone for the SYNT001 clinical development program and highlights the high unmet medical need for new therapies with the potential to improve the lives of pemphigus patients and their families,” said Jean-Paul Kress, MD, president and CEO of Syntimmune. “We believe there is significant potential for SYNT001 in pemphigus and other autoimmune diseases and look forward to providing additional clinical data.”
Positive preliminary results from a phase 1b proof-of-concept trial of SYNT001 in patients with pemphigus served as the basis for the approval. In the first cohort, SYNT001 was observed to be well tolerated and induced a rapid reduction in IgG and circulating immune complex levels. In addition, as measured by Pemphigus Disease Area Index (PDAI) score, SYNT001 induced clinical improvement, with clinical effect persisting beyond the treatment period.