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FDA reverses course on Regenxbio’s childhood gene therapy after rejection

The FDA will reconsider Navsunli, a one-time gene therapy for Hunter syndrome also known as MPS II. The agency reversed its previous demand for an untreated control arm and now backs an accelerated approval path. Regenxbio plans to resubmit its BLA in Q3 2026.

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What changed

The FDA dropped placebo requirements and agreed to a path for accelerated approval after rejecting the drug four months ago.

Live updates

  1. FDA Reverses Rejection of Regenxbio Hunter Syndrome Gene Therapy

    The FDA will reconsider Navsunli, a one-time gene therapy for Hunter syndrome also known as MPS II. The agency reversed its previous demand for an untreated control arm and now backs an accelerated approval path. Regenxbio plans to resubmit its BLA in Q3 2026.

    What's confirmed:

    • The FDA reversed its rejection of Regenxbio's gene therapy for Hunter syndrome.
    • Navsunli is a one-time gene therapy for Mucopolysaccharidosis II, also known as Hunter syndrome.
    • The FDA dropped the requirement for an untreated control arm or placebo.
    • Regenxbio expects to resubmit the BLA in Q3 2026.
    • This is the third rare disease drug reversal by the FDA in two months.
    • The FDA confirmed that no additional studies are required.
    confidence 100%