FDA Grants Priority Review to Orphazyme’s Niemann-Pick Disease Drug

Rare Daily Staff

The U.S. Food and Drug Administration has accepted Orphazyme’s application to market its experimental drug arimoclomol for the treatment of Niemann-Pick Disease type C, a rare and deadly lysosomal storage disorder.

NPC is a genetic, progressively debilitating, fatal neurovisceral disease. As a consequence of a genetic mutation, patients with the condition produce a misfolded NPC protein that prevents the body from clearing certain lipids. As these lipids build-up in tissues and organs, including the brain, it causes progressive damage. The estimated prevalence of NPC in the United States and Europe combined is 1,000-2,000. There are no approved treatments for NPC in the United States and only one approved product in Europe.

Arimoclomol amplifies the production of heat-shock proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally, and crosses the blood brain barrier. It has been studied in seven phase 1 and three phase 2 trials. Arimoclomol is in clinical development for NPC, Gaucher disease, sIBM, and ALS.

The FDA grants Priority Review to applications for potential therapies that, if approved, could offer a significant improvement in safety or effectiveness, diagnosis, or prevention of serious conditions. This designation shortens the review period from the standard 10 months to six months from the acceptance of the NDA. The FDA has set a target action date of March 17, 2021 under the Prescription Drug User Fee Act. The agency indicated that it does not plan to hold an advisory committee meeting to discuss the application.

“Acceptance of the arimoclomol filing by the FDA is another major step forward in the effort to bring a treatment to people affected by Niemann-Pick disease Type C and is a reflection of the commitment of the entire community coming together to support promising research,” said Joslyn Crowe, executive director of the National Niemann-Pick Disease Foundation.

Arimoclomol has been granted FDA Fast Track and Breakthrough Therapy designations for NPC, as well as Orphan Drug and Rare Pediatric Disease designations.

Orphazyme expects to file an application for marketing approval for the drug in Europe in before year end.

Photo: Joslyn Crowe, executive director of the National Niemann-Pick Disease Foundation