Orphazyme has recently submitted their New Drug Application to the FDA for their Niemann-Pick disease type C (NPC) treatment, arimoclomol. This application comes after the biopharmaceutical company was already granted the Fast Track, Orphan Drug, Rare Pediatric Disease, and Breakthrough Therapy designations.
About Niemann-Pick Disease Type C
Niemann-Pick disease is a group of rare and severe lipid storage disorders. It occurs when the body is unable to transfer sphingomyelin, a type of lipid, into the cells, causing them to accumulate in the lysosomes. This disease is divided into three types: A, B, and C. Between these types, severity, onset, and symptoms vary. Types A and B of this disease occur when there are mutations in the SMPD1 gene, which affects the activity of sphingomyelin. Type C is caused by mutations in the NPC1 or NPC2 genes, which are responsible for a protein that transports sphingomyelin. These genes are all inherited in an autosomal recessive pattern.
Symptoms of Niemann-Pick disease vary depending on the type and location of the accumulation of sphingomyelin. The liver and spleen are common places in which sphingomyelin accumulates, and symptoms would be a loss of appetite, an enlarged abdomen, low levels of platelets in the blood, and pain. If the central nervous system is affected, symptoms may be slurred speech, difficulty swallowing, impaired eye movements, and the loss of intellectual abilities. Bones are not as commonly affected, but when they are, people experience sleep related disorders and enlarged bone marrow cavities. Type C, which is the form treated by arimoclomol, is characterized by neurological abnormalities, like loss of coordination and difficulty speaking. Difficulty in swallowing is also common. Intellectual disabilities worsen over time. Cataplexy and seizures may occur as the disease progresses as well.
Treatment for this disease is dependent on the type, but they are all focused on managing symptoms. Anti-seizure, sleep-inducing, and anti-depression medications may all be prescribed. There are also many treatments in various phases of research, with Orphazyme’s treatments being an example.
Arimoclomol for NPC
Now that the application has been submitted, the FDA has 60 days to accept it for review. Even though the decision is in the hands of the FDA now, Orphazyme is still working on another application. They expect to submit their Marketing Authorization Application to the European Medicines Agency in the second half of this year.
Both of these applications were made possible after positive results from randomized and controlled clinical trials. It was proven to elevate Heat-Shock protein levels and make effective changes in biomarkers. In fact, this data suggests that arimoclomol may be able to treat other lysosomal disorders as well.
Arimoclomol is important to those with NPC as there are currently no disease modifying treatments. If approved, this therapy could greatly better the lives of affected individuals. The National Niemann-Pick Disease Foundation is very excited about the NDA, and they hope that it gains FDA approval.
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