Neuralstem, Inc. (Nasdaq:CUR), a biopharmaceutical company focused on
the development of nervous system therapies based on its neural stem
cell technology, today announced that the U.S. Food and Drug
Administration has granted orphan drug designation to NSI 189 powder for the treatment of Angelman syndrome.
“Angelman Syndrome is a rare disease with significant unmet medical
need, and for which there are no FDA-approved therapies,” said Jim
Scully, Chief Executive Officer of Neuralstem. “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.”
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.
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. Orphan drug designation confers
seven years of market exclusivity upon FDA approval, as well as other
development incentives, such as tax credits related to clinical trial
expenses, an exemption from the FDA-user fee and FDA assistance in
clinical trial design.
About Angelman Syndrome
Angelman Syndrome (AS) is a rare congenital genetic disorder caused by a
lack of function in the UBE3A gene on the maternal 15th chromosome. It
affects approximately one in 15,000 people - about 500,000 individuals
globally. Symptoms of AS include developmental delay, lack of speech,
seizures, and walking and balance disorders. Patients with AS may never
walk or speak and require life-long care. Life expectancy is normal
which places a significant burden on patients and caregivers. There are
currently no FDA-approved therapies for the treatment of Angelman
syndrome.
