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REGENXBIO Announces Expansion of AFFINITY DUCHENNE® Trial to Include a New Cohort of Younger Patients

Company to begin enrollment of patients aged 1-3 years

Expects data from younger cohort to be part of pivotal plans and BLA filing for broad label

End-of-Phase II meeting with FDA scheduled for late July to finalize pivotal program design

Based on recent commercial landscape, confirmed accelerated approval pathway remains available given ongoing unmet need and RGX-202 differentiated design

Remains on track to initiate pivotal trial in late Q3 to early Q4 2024

The next potential therapy to become available for Duchenne patients

REGENXBIO announced they are expanding their AFFINITY DUCHENNE Trial, which delivers a micro-dystrophin via AAV8, to include a new cohort of younger patients. The trial, which has been enrolling ambulatory boys with Duchenne aged 4 to 11, is now expected to enroll up to five individuals aged 1-3 as well. These younger participants will receive RGX-202 at the pivotal dose level (dose level 2, at 2×1014 genome copies/kg body weight). REGENXBIO expects to share initial strength and functional assessment data for individuals already treated (at both dose levels) later this year.

Press Release here

The post REGENXBIO Announces Expansion of AFFINITY DUCHENNE® Trial to Include a New Cohort of Younger Patients appeared first on CureDuchenne.

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