REGENXBIO announced the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation to RGX-181. RGX-181 is a one-time treatment candidate for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease caused by mutations in the tripeptidyl peptidase 1 (TPP1) gene. In addition to the Rare Pediatric Disease Designation, REGENXBIO previously received Orphan Drug Designation from the FDA for RGX-181.
The FDA grants Rare Pediatric Disease Designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. If a new biologics license application (BLA) for RGX-181 is approved, REGENXBIO may be eligible to receive a priority review voucher, which can be redeemed to obtain priority review for any subsequent marketing application and may be sold or transferred. This program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.
"There is an urgent need for treatment options for CLN2 disease, a serious and life-threatening disease, which is emphasized by RGX-181 receiving Rare Pediatric Disease Designation, in addition to Orphan Drug Designation," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "We believe that RGX-181 administered as a one-time treatment can potentially correct the underlying genetic condition and halt the progression of this devastating disease. We look forward to filing an IND with the FDA for the first-in-human clinical trial in the second half of 2019."
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RGX-181 is designed to use REGENXBIO's 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. REGENXBIO plans to submit an Investigational New Drug (IND) application for RGX-181 to the FDA in the second half of 2019 to enable initiation of a first-in-human clinical trial.
RGX-181 is being developed as a novel, one-time treatment for CLN2 disease utilizing the NAV AAV9 vector to deliver the gene encoding for 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 an animal model for CLN2 disease, treatment with RGX-181 has been shown to restore TPP1 activity to levels greater than those in non-affected animals, and to improve neurobehavioral function and survival. 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.
Late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a form of Batten disease, is a rare, pediatric-onset, autosomal recessive, neurodegenerative lysosomal storage disorder caused by mutations in the TPP1 gene. Mutations in the TPP1 gene, and subsequent deficiency in TPP1 enzymatic activity, result in lysosomal accumulation of storage material and degeneration of tissues including the brain and retina. CLN2 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. Onset of symptoms is generally between two to four years of age with initial features of recurrent seizures (epilepsy), language delay, and difficulty coordinating movements (ataxia). There is currently no cure for CLN2 disease. Current treatment options include palliative care or enzyme replacement therapy, wherein recombinant TPP1 is administered into the lateral ventricles via a permanently implanted device on a biweekly basis.