RegenxBio product RGX-181 has been granted FDA Rare Pediatric Disease Designation for Batten Disease Condition known as CLN2

FDA Granted Rare Pediatric Disease Designation for RegenxBio’s Product RGX-181 for CLN2


RegenxBio (RGNX), a development-stage firm that focuses on designing and developing gene therapy based on its proprietary NAV® Technology Platform, announced that the U.S. FDA granted Rare Pediatric Disease Designation to its RGX-181 therapy.

RegenxBio is a leading clinical-stage biotechnology company that promises to cure genetic-derived diseases with its gene therapy. RegenxBio’s NAV Technology Platform is a proprietary adeno-associated virus (AAV) gene delivery platform which consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. 

RegenxBio and its licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas. 

About 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.

About RGX-181

A one-time treatment candidate for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease; one of the most common forms of Batten disease. The FDA granted RGX-181 also an Orphan Drug Designation.

The product is designed to use RegenxBio’s NAV AAV9 vector to deliver the TPP1 gene directly to the central nervous system (CNS) which is expected to induce sustained levels of TPP1, the deficient enzyme in children with the CLN2.  Following a single administration, given by intracisternal injection, the RGX-181 treatment will provide a durable source of TPP1 and allow for long-term correction of cells throughout the CNS

The FDA grants Rare Pediatric Disease Designation for serious and life-threatening diseases that primarily affect children ages 18 years and younger and fewer than 200,000 people in the United States. With this designation RegenxBio may be eligible to receive a priority review voucher if a new biologics license application (BLA) for RGX-181 is approved. 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.

A Quote from RegenxBio’s President & CEO

Kenneth T. Mills, President and Chief Executive Officer of RegenxBio said, “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. 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.”

Prohost Observations

The extent of CNS correction observed, with the use of RegenxBio’s product RGX-181 in preclinical animal study results, implies that the products could represent a suitable therapeutic option for patients with the CLN2 condition. A successful outcome in human clinical trials, if materialized and we believe it will, would spare the children the neurodegenerative lysosomal storage disorder’s complications, including seizures, rapid deterioration of motor functions, cognitive decline, loss of vision and blindness and premature death by mid-childhood.

Our optimism is based on the firm’s history of accomplishments, of the licensees of its NAV Technology Platform and of the successful clinical trial results with a product that the firm had licensed to AveXis which was acquired by Novartis. The product is expected to be granted FDA approval soon for Spinal Muscular Atrophy (SMA).  

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.

We are optimistic.

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