The US FDA has awarded Orphan Drug status to the investigational drug NSI-189 for use by people with Angelman syndrome. You can read the source article here, at Nasdaq.
About Angelman Syndrome
Angelman syndrome is a genetic condition that causes lifelong physical and intellectual disability. It has been linked to genetic changes in Chromosome 15. People with the condition usually have a normal lifespan but are likely to need to be looked after throughout their life. According to the NHS, typically, the first signs of the condition begin when children reach around six to twelve months old. When children with the condition get older, they may not be able to speak, or only be able to use a few words. However, some children with Angelman syndrome can communicate in other ways, such as through gestures. People with Angelman syndrome are also likely to have a movement or balance disorder, and behavioural traits such as a happy demeanour and excitability, amongst other features.
NSI-189 is an investigational small molecule drug. Researchers have tested the drug in pre-clinical studies of mouse models of Angelman syndrome, and it was found to restore long-term potentiation. This is a measure of synaptic plasticity, which has been linked to memory.
In addition to Angelman syndrome, NSI-189 is also being explored as a potential drug for major depressive disorder, Type 1 and Type 2 diabetes, irradiation-induced cognitive impairment, and stroke.
About Orphan Drug Designation
A spokesperson for Neuralstem, the company developing the drug, says that the FDA’s decision to grant NSI-189 Orphan Drug status is “an important regulatory milestone.”
Orphan Drug designation is designed to support the development process of drugs for conditions that are considered ‘rare’, defined as affecting less than 200,000 people within the US. The designation makes the developers eligible for several benefits, such as seven years of marketing exclusivity, tax credits for clinical trials, assistance in designing clinical trials, and exemptions from certain fees. More information about the FDA’s Orphan Drug designation can be found at their website, here.