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Exclusive | FDA Gives Third Rare-Disease Drug Another Shot, Regenxbio Says

The FDA allowed Regenxbio to resubmit its application for the accelerated approval of NAVSUNLI. This therapy targets Mucopolysaccharidosis Type II. The agency's shift in evidence requirements for rare disease drugs may also benefit other biotech firms.

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What changed

New reports indicate that uniQure also received a similar FDA reversal for a gene therapy.

Live updates

  1. FDA Reverses Rejection of Regenxbio Hunter Syndrome Therapy

    The FDA allowed Regenxbio to resubmit its application for the accelerated approval of NAVSUNLI. This therapy targets Mucopolysaccharidosis Type II. The agency's shift in evidence requirements for rare disease drugs may also benefit other biotech firms.

    What's confirmed:

    • The FDA reversed its rejection of Regenxbio's gene therapy for Hunter syndrome.
    • Regenxbio can resubmit its application for accelerated approval.
    • The FDA also cleared uniQure to file or refile for approval of a gene therapy.
    • Regenxbio will receive $100 million from AbbVie after dosing the first patient in the Phase IIb/III NAAVIGATE trial for diabetic retinopathy.

    Still unconfirmed:

    • The FDA's updated outlook on rare disease evidence could benefit Skyhawk Therapeutics, Capricor Therapeutics, and Biohaven.
    • An unnamed 79-year-old man received early access to an Eli Lilly drug.
    confidence 100%

  2. FDA Reverses Course on Regenxbio's Rare-Disease Gene Therapy

    The FDA has reversed its rejection of Regenxbio's gene therapy for Hunter syndrome, allowing the company to resubmit its application for accelerated approval. This is the latest in a series of reversals for rare disease treatments. Regenxbio expects to meet with the FDA in July and resubmit its Biologics License Application (BLA) in Q3 2026. The therapy, called NAVSUNLI, is a potential treatment for Mucopolysaccharidosis Type II (MPS II).

    What's confirmed:

    • The FDA will reconsider Regenxbio's gene therapy for Hunter syndrome.
    • Regenxbio will resubmit its Biologics License Application (BLA) for NAVSUNLI in Q3 2026.
    • The FDA's reversal allows for an accelerated approval bid for NAVSUNLI.
    • Regenxbio expects to meet with the FDA again in July 2026.

    Still unconfirmed:

    • The FDA's change in stance could have immediate benefits for other rare disease applications.
    confidence 50%