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# FDA Priority Review for Niemann-Pick Disease Drug
- URL: https://www.fdaweb.com/fda-priority-review-for-niemann-pick-disease-drug/
- Published: 2020-09-17T12:00:00.000Z
- Updated: 2026-09-14T16:29:01.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5147677

FDA has accepted for priority review a CytRx NDA for arimoclomol for treating Niemann-Pick disease Type C. CytRx says that arimoclomol works by amplifying the production of heat-shock proteins, which can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. The agency has set a review action target date of 3/17/2021.

Niemann-Pick disease Type C is a rare, genetic and progressive disease that impairs the ability of the body to move cholesterol and other fatty substances (lipids) inside the cells, the company says. “The result is an accumulation of lipids within the body’s tissue, including the brain tissue, causing damage to the affected areas,” it says. Symptoms are more common in infancy and childhood and the rate of progression is usually much slower in individuals with symptom onset during adulthood.