The FDA designates Arcturus Therapeutics’ (NASDAQ:ARCT) lead candidate ARCT-810 an Orphan Drug for the treatment of ornithine transcarbamylase deficiency (OTCD), a relatively rare urea cycle disorder in which ammonia accumulates in the body leading to nervous system damage.
ARCT-810 is an mRNA construct designed to deliver OTC to liver cells.
The company plans to file an IND in Q1 2020.
Among the benefits of Orphan Drug status in the U.S. is a seven-year period of market exclusivity for the indication, if approved.
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