FDA Grants RegenxBio Rare Pediatric Disease Designation for Gene Therapy for CLN2 Form of Batten Disease

January 31, 2019

Rare Daily Staff

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to RegenxBio’s RGX-181, an experimental gene therapy in development to treat for late-infantile neuronal ceroid lipofuscinosis type 2 disease, one of the most common forms of Batten disease.

Late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease is a rare, neurodegenerative lysosomal storage disorder caused by a deficiency in the enzyme TPP1, which result in accumulation of metabolic waste 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. Current treatment options include palliative care or enzyme replacement therapy, which is administered into the brain through a permanently implanted device on a biweekly basis.

RGX-181 is designed to use RegenxBio’s NAV AAV9 vector to deliver the TPP1 gene directly to the central nervous system, which may induce sustained levels of TPP1, the enzyme deficient in children with CLN2 disease. The company plans to submit an application to begin human clinical trials for RGX-181 to the FDA in the second half of 2019.

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 for RGX-181 is approved, RegenxBio may be eligible to receive a Priority Review voucher.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Ultragenyx and Kyowa Hakko Kirin sold their Priority Review voucher for $80.6 million.

The company previously received Orphan Drug designation from the FDA for RGX-181.

“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 Mills, president and CEO 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.”


January 31, 2019
Photo: Kenneth Mills, president and CEO of RegenxBio



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