FDA accepts Orphazyme arimoclomol application for rare lipid disorder

Under Priority Review status, the FDA accepts Orphazyme A/S’s (ORPH) marketing application seeking approval for lead drug arimoclomol for the treatment of Niemann-Pick disease Type C, a rare inherited disorder in which the body is unable to transport cholesterol and other lipids inside of cells. The fatty substances accumulate in various body tissues, leading to potentially life-threatening nerve and organ damage.

The agency’s action date is March 17, 2021. No advisory committee meeting is planned.

Orally administered arimoclomol amplifies the production of heat-shock proteins, which can rescue defective misfolded proteins, clear protein aggregates and improve the function of lysozymes, naturally occurring enzymes that function as antimicrobial agents.

The company secured the rights to arimoclomol from CytRx in 2011.

https://seekingalpha.com/news/3614460-fda-accepts-orphazyme-arimoclomol-application-for-rare-lipid-disorder