Nanoscope Therapeutics, an Arlington-based clinical-stage biotech company developing ocular therapies, said it has won a special designation from the federal Food and Drug Administration for a drug it’s developing.
Nanoscope said it received an “Orphan” drug designation for a gene-therapy based treatment of Stargardt disease, a form of inherited retinal degenerative disease caused by gene mutation and passed onto children. The FDA grants Orphan designations to drugs and biologics under development that promise to treat rare conditions and illnesses. Such designations allow significant incentives, such as fee waiver for FDA applications.
Nanoscope is one of two TechFW incubator clients to announce this week it won an Orphan drug designation from the FDA. AyuVis announced it won such a designation for a drug it’s developing to treat a rare and potentially fatal pulmonary condition in premature babies. "Currently, there are no approved therapies for Stargardt Disease" Sulagna Bhattacharya, chief executive of Nanoscope, said in a release.
According to the Federation for Fighting Blindness, Stargardt disease is the most common form of inherited macular degeneration, affecting about 30,000 people in the U.S., Nanoscope said. “The loss of vision is devastating to both children and adults, and significantly impacts their quality of life. There is an immense need to restore vision in these patients.”
It’s the second Orphan designation Nanoscope has received for one of its therapies.