The U.S. Food and Drug Administration (FDA) has granted a Priority Review to assess arimoclomol to treat children with Niemann-Pick disease Type C.
A Priority Review means that the FDA will review the drug within 6 months instead of the standard 10 months. A Prescription Drug User Fee Act (PDUFA) date of March 17, 2021 has been set.
Niemann-Pick disease type C is a rare lysosomal storage disorder in which toxic quantities of lipids accumulate in the spleen, liver, lungs, bone marrow, and brain. Symptoms may include lack of muscle coordination, brain degeneration, learning problems, loss of muscle tone, increased sensitivity to touch, spasticity, feeding and swallowing difficulties, slurred speech, and an enlarged liver and spleen.
Arimoclomol is an orphan drug that amplifies the production of heat-shock proteins and thereby rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes.
Until recently, the outlook for arimoclomol to treat Niemann-Pick disease type C was looking grim. In 2019, a phase III clinical trial failed to meet its primary endpoint but a sub-group analysis as well as an open-label extension study did show the drug to be effective. In July 2020, the developers of the drug, Orphazyme, began a rolling NDA submission for the drug and on September 16, 2020, the company announced that the FDA had accepted the application and granted a Priority Review of the drug.
The FDA has also granted arimoclomol with a Rare Pediatric Disease Designation. If approved for this designation, the company will receive a Priority Review Voucher that can be used for future FDA applications (or sold to another company for millions of dollars).
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