Neuralstem Inc (NASDAQ:CUR) shares got a jolt to higher territory on Thursday after the US Food and Drug Administration granted orphan drug approval for its drug treatment of a rare disease called Angelman syndrome.
The drug is called NSI-189 and orphan drug designation gives companies special status and incentives to encourage the development of drugs for diseases affecting fewer than 200,000 people in the US.
Neuralstem shares gained 6.3% to trade at US$1.18, having hit a peak of US$1.35.
According to the Mayo clinic, Angelman syndrome is a genetic disorder that causes developmental disabilities, neurological problems and, sometimes, seizures. It is caused by a genetic mutation called UBE3A. Normally, people inherit one copy of the gene from each parent. In Angelman, part of the maternal copy is missing or damaged.
People with Angelman syndrome often smile and laugh frequently, and have happy, excitable personalities, the clinic said. Developmental delays, between about six and 12 months of age, are usually the first signs of Angelman syndrome. Seizures often begin between the ages of two and three years old.
People with Angelman syndrome tend to live a normal life span, but the disease cannot be cured. Treatment focuses on managing medical and developmental issues.
Neuralstem is based in Germantown, Maryland. It is a biopharmaceutical company focused on the development of nervous system therapies based on its neural stem cell technology.