Biotechnology company REGENXBIO Inc (Nasdaq:RGNX) reported on Wednesday the receipt of the US Food and Drug Administration's (FDA) orphan drug designation for the one-time treatment candidate RGX-181 for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease.

Late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a form of Batten disease, is characterized by seizures, rapid deterioration of language and motor functions, cognitive decline, loss of vision and blindness, and premature death by mid-childhood.

The company said RGX-181 is designed to use its NAV AAV9 vector to deliver the TPP1 gene directly to the central nervous system (CNS), which may induce sustained levels of TPP1, the enzyme deficient in children with CLN2 disease. Following a single administration given by intracisternal injection, RGX-181 treatment is designed to modify cells in the CNS, thereby providing a durable source of TPP1 and allowing for long-term correction of cells throughout the CNS.

In 2019, the company intends to submit an Investigational New Drug (IND) application for RGX-181 to the US FDA to enable initiation of a first-in-human clinical trial.

Additionally, the extent of CNS correction observed in animal studies suggests that RGX-181 has the potential to be an important and suitable therapeutic option for patients with CLN2 disease, concluded the company.