Regenxbio Inc. (NASDAQ:RGNX) on Monday said it aligned with the Food and Drug Administration (FDA) regarding the next steps needed for a potential accelerated approval of Navsunli (RGX-121, clemidsogene lanparvovec).
The potential one-time gene therapy is seeking approval for Mucopolysaccharidosis II (MPS II), an ultra-rare neurodegenerative disease also known as Hunter syndrome.
The Wall Street Journal was the first to report it, highlighting another policy shift at the agency under its new leadership.
FDA Reverses Rejection Of Regenxbio’s Gene Therapy
The FDA acknowledged that the existing Navsunli data are sufficient to be considered for the accelerated approval pathway and that the company does not need to enroll additional patients or conduct additional studies, including the FDA’s previously recommended incorporation of an untreated control arm.
Regenxbio expects the Type A meeting to take place in July and to resubmit the BLA rapidly following the meeting in the third quarter of 2026.
In February, the FDA rejected the gene therapy for Mucopolysaccharidosis II (MPS II), an ultra-rare neurodegenerative disease also known as Hunter syndrome.
The reversal represents the latest instance of the agency revisiting decisions made under former FDA vaccines and biotechnology drugs division head Vinay Prasad, particularly regarding clinical trial requirements for therapies targeting rare diseases.
FDA Eases Position On Rare Disease Trial Requirements
Drugmakers developing therapies for ultra-rare conditions have argued that placebo requirements are often impractical because patient populations are extremely small, with some disorders affecting only a few dozen individuals annually.
Industry participants have also maintained that such requirements can significantly extend development timelines.
Regenxbio Appeals Rejection After Meeting With FDA Leadership
The WSJ on Monday reported that Regenxbio executives met with then-FDA Commissioner Marty Makary in April to discuss the agency’s rejection of Navsunli.
During those discussions, Makary encouraged the company to formally appeal the decision, assuring executives that the application would receive a review conducted with "fresh eyes," Simpson said.
FDA Path Forward For Resubmission
In May, Replimune Group Inc. (NASDAQ:REPL) said that the company and the FDA have aligned on a path forward for resubmission and reconsideration of the Biologics License Application (BLA) for RP1 (vusolimogene oderparepvec) in combination with nivolumab for advanced melanoma.
The company will resubmit the RP1 BLA in the coming days.
Last week, uniQure N.V. (NASDAQ:QURE) also shared plans for a Biologics License Application (BLA) submission for AMT-130, a gene therapy treatment for Huntington’s disease.
RGNX Stock Price Activity: Regenxbio shares were up 13.33% at $8.84 during premarket trading on Monday, according to Benzinga Pro data.
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