REGENXBIO Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RGX-121 Gene Therapy for Hunter Syndrome
- RMAT recognizes that the preliminary clinical evidence from RGX-121, a potential one-time AAV Therapeutic, indicates the potential to address unmet medical needs for MPS II
- RMAT designation is for gene therapies intended to treat or cure serious condition in order to expedite the drug development and review processes
- CAMPSIITE™ trial is enrolling MPS II patients as part of a pivotal program that incorporates material from the NAVXpress™ platform process manufactured at the REGENXBIO Manufacturing Innovation Center and continues to support plan to file Biologics License Application in 2024 using the accelerated approval pathway
"We are pleased that the FDA has granted RMAT designation for RGX-121 for its potential to address the neurological manifestations of MPS II and prevent or stabilize cognitive decline, which remains a significant unmet need for this community," said
Established under the 21st Century Cures Act, a drug is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition.
RMAT designation includes the benefits of early FDA interactions to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the BLA and other opportunities to expedite development and review. In addition, sponsors of products that have been granted RMAT designation and which received accelerated approval may be able to fulfill the post-approval requirements from clinical evidence obtained from sources other than the traditional confirmatory clinical trials.
"Severe Hunter syndrome is a progressively debilitating disorder that affects a child's physical and mental development and leads to a shortened lifespan, and I applaud the FDA for granting RMAT designation to RGX-121," said
"We know that families are waiting for new treatment options for this serious disease and that every day matters," said
RGX-121 Program Highlights
The Phase I/II/III CAMPSIITE™ trial of RGX-121 for MPS II patients aged 4 months up to 5 years is ongoing and in
A Phase I/II trial of RGX-121 for the treatment of pediatric patients with MPS II over the age of five years old is also ongoing.
RGX-121 is designed to use the NAV® AAV9 vector to deliver the human iduronate-2-sulfatase gene (IDS) which encodes the iduronate-2-sulfatase (I2S) enzyme to the central nervous system (CNS). Delivery of the IDS gene within cells in the CNS could provide a permanent source of secreted I2S beyond the blood-brain barrier, allowing for long-term cross correction of cells throughout the CNS. RGX-121 has received Orphan Drug Product, Rare Pediatric Disease, Fast Track and Regenerative Medicine Advanced Therapy designations from the
About Mucopolysaccharidosis Type II (MPS II)
MPS II, or Hunter Syndrome, is a rare, X-linked recessive disease caused by a deficiency in the lysosomal enzyme iduronate-2-sulfatase (I2S) leading to an accumulation of glycosaminoglycans (GAGs), including heparan sulfate (HS) in tissues which ultimately results in cell, tissue, and organ dysfunction, including in the central nervous system (CNS). MPS II is estimated to occur in 1 in 100,000 to 170,000 births. In severe forms of the disease, early developmental milestones may be met, but developmental delay is readily apparent by 18 to 24 months. Specific treatment to address the neurological manifestations of MPS II remains a significant unmet medical need. Key biomarkers of I2S enzymatic activity in MPS II patients include its substrate heparan sulfate (HS) D2S6, which has been shown to correlate with neurocognitive manifestations of the disorder.
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