05:35 PM EDT, 04/15/2024 (MT Newswires) -- Zevra Therapeutics ( ZVRA ) disclosed late Monday new data from the arimoclomol expanded access program to treat Niemann-Pick disease type C, an inherited metabolic disorder, demonstrated a "clinically meaningful slowing of disease progression."

Chief Executive Neil McFarlane said the company will continue collaborating with the US Food and Drug Administration as it reviews arimoclomol's new drug application ahead of the Prescription Drug User Fee Act date on Sept. 21.

Adults treated with arimoclomol, including those with and without miglustat use, generally had a "stable disease course" over two years of treatment and follow-up.

The safety profile was "consistent" with that observed in the phase 2/3 study.