Regenxbio completes its BLA filing for first potential gene therapy for MPS II
US biotech Regenxbio (Nasdaq: RGNX) has successfully completed the BLA submission for its mucopolysaccharidosis II (MPS II) RGX-111 and is on track for potential US Food and Drug Administration (FDA) approval via accelerated approval in second-half 2025.
The firm’s shares edged up 2.6% to $6.72 on the news, which came alongside its 2024 financial results. It reported a net loss of $51.2 million, or $1.01 basic and diluted net loss per share, for the three months ended December 31, 2024, compared to a net loss of $62.9 million, or $1.43 basic and diluted net loss per share a year earlier.
The company says RGX-121 remains on track to be the potential first gene therapy and one-time treatment approved for MPS II, also known as Hurler syndrome, adding that Regenxbio also continues to advance RGX-111 for MPS I through the recent strategic partnership with Japan’s Nippon Shinyaku (TYO: 4516).
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