Results support BLA submission in 2024 using the accelerated approval pathway Primary endpoint of patients achieving reduction in CSF biomarker of MPS II.
REGENXBIO Announces Pivotal Trial of RGX-121 for the Treatment of MPS II Achieves Primary Endpoint streetinsider.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from streetinsider.com Daily Mail and Mail on Sunday newspapers.
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. | May 23, 2023
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Sunday, February 28, 2021, was Rare Disease Day. With so much focus on COVID-19 throughout 2020, it’s important to recognize the continued work done in rare disease drug development by sponsors and FDA throughout 2020. In addition, a number of policies implemented in response to COVID-19 are expected to have a positive impact on rare disease drug development going forward. Yet, the lasting nature of these policies, post-pandemic, remains uncertain, creating an opportunity for rare disease drug sponsors to be proactive in engaging with regulatory authorities and the patient community to advocate the retention of these positive policy developments.