Niemann-Pick Disease Type C is a lysosomal storage disorder caused by malfunction of the NPC1 protein. This protein’s dysfunction results in a buildup of lipids in the lysosomes. As a result, cells begin to die, eventually resulting in organ failure. While prognosis varies between patients, the average lifespan is about ten years.
The disease is progressive, and as the patient ages, their quality of life significantly decreases. Not only are these patients living lives that are much too short, they are dealing with a continuous decrease in cognitive and motor function along the way.
We need more options for these patients, and we need them fast.
Thankfully, IB1000s, a compound series created by IntraBio was just granted Orphan Drug Designation by the FDA. This is a positive step toward achieving FDA approval for the treatment.
What led to this designation?
IntraBio has been working on this development for quite some time. They have evaluated IB1000s in numerous studies utilizing a total of 175 patients. Their work has shown that the investigation of 1B1000s is warranted for at least 18 different conditions. These include lysosomal storage disorders like Niemann-Pick as well as neurodegenerative diseases.
1B1000s had already been awarded orphan medicinal drug designation by the European Commission.
IntraBio is working on developing clinical trials for the treatment. They hope to conduct studies across the world and are currently working on applications for multi-national, multi-center trials.
The hope, is that successful trials for Niemann-Pick disease will not only help those patients, but will prove the treatment’s potential for other conditions. These include Multiple Sclerosis and ALS.
The granting of Orphan Drug Designation for this treatment shows the medical worlds commitment to researching Niemann-Pick and other rare conditions. It’s evident we need faster progress for rare disease patients, and this is just one more indication we’re moving in the right direction.
You can read more about this new designation here.