Neuralstem receives US FDA orphan drug designation for treatment of Angelman Syndrome

KUALA LUMPUR, Aug 10 (Bernama) -- Neuralstem Inc -- a biopharmaceutical company focused on the development of nervous system therapies -- received orphan drug designation  from the U.S. Food and Drug Administration (FDA) to NSI-189 for the treatment of Angelman Syndrome (AS).

“Angelman Syndrome is a rare disease with significant unmet medical need and for which there are no FDA-approved therapies,” said chief executive officer of Neuralstem, Jim Scully.

“Orphan drug designation is an important regulatory milestone in the development of NSI-189 and we are committed to evaluating NSI-189’s role as a treatment to improve the lives of patients with Angelman syndrome,” Scully added.

In pre-clinical models, NSI-189 has demonstrated the ability to restore long term potentiation (LTP) -- a measure of synaptic plasticity and an in vitro biomarker of memory, a statement said.

The FDA's orphan-drug designation program provides special status and incentives to encourage the development of drugs for diseases affecting fewer than 200,000 people in the U.S.

It also confers seven years of market exclusivity upon FDA approval, as well as other development incentives such as tax credits related to clinical trial expenses, exemption from the FDA-user fee and FDA assistance in clinical trial design.

Angelman Syndrome is a rare congenital genetic disorder that affects approximately one in 15,000 people - about 500,000 individuals globally.  Symptoms of AS include developmental delay, lack of speech and seizures while patients may never walk or speak and require life-long care.

-- BERNAMA