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Regenxbio Shares Rise on Plans to Resubmit Navsunli BLA for MPS II
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Key Takeaways
Regenxbio said FDA aligned on next steps for potential accelerated approval of Navsunli in MPS II.
FDA acknowledged existing clinical data are sufficient to support accelerated approval of Navsunli.
Following a Type A meeting, the BLA for Navsunli in MPS II is planned for resubmission in Q3 2026.
Regenxbio (RGNX - Free Report) announced that it has reached alignment with the FDA on the next regulatory steps for the potential accelerated approval of Navsunli (clemidsogene lanparvovec-sngl, RGX-121), a one-time investigational gene therapy being developed to treat patients with Mucopolysaccharidosis II (MPS II) or Hunter syndrome.
Shares of the company were up 16.3% yesterday following the announcement of the news.
Following recent constructive discussions, the FDA has now acknowledged that the existing clinical data are sufficient to support accelerated approval of Navsunli for MPS II, an ultra-rare neurodegenerative disease.
The regulatory body has asked the company to request a Type A meeting to review existing longer-term biomarker and clinical data, after which the company plans to resubmit the biologics license application (BLA) for Navsunli in the third quarter of 2026.
The Type A meeting is expected to be held in July next month.
RGNX’s Price Performance
Year to date, shares of Regenxbio have declined 37% compared with the industry’s decrease of 1.1%.
Image Source: Zacks Investment Research
RGNX’s Recent Developments With Navsunli
In February 2026, the FDA issued a complete response letter (CRL) to the BLA for Navsunli in MPS II.
Back then, the CRL outlined several possible paths forward, such as conducting a new study, collecting longer-term data from additional patients, or using an untreated control group.
Last month, the FDA lifted the clinical hold for Navsunli.
The FDA accepted the BLA for Navsunli under the accelerated approval pathway in May 2025.
Hunter syndrome is caused by a deficiency of the iduronate 2-sulfatase enzyme, leading to the buildup of harmful substances in tissues, including the brain, and resulting in progressive cognitive, motor and organ damage.
In March 2026, the FDA granted accelerated approval to Denali Therapeutics’ (DNLI - Free Report) tividenofusp alfa-eknm, under the brand name Avlayah, for the treatment of Hunter Syndrome. The continued approval for this indication may be contingent upon verification of clinical benefit in a confirmatory study.
Developed by Denali, Avlayah is enabled by its TransportVehicle platform, which facilitates delivery of biologics throughout the body, including the brain.
DNLI’s Avlayah is an enzyme replacement therapy indicated for pediatric patients with Hunter syndrome, targeting neurological symptoms when initiated early.
Over the past 60 days, estimates for Immunocore’s 2026 bottom line have improved from a loss of 88 cents per share to earnings of 6 cents. Over the same period, EPS estimates for 2027 have risen from 24 cents to 87 cents. IMCR stock has lost 15.4% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 46.66%.
Over the past 60 days, estimates for Liquidia’s 2026 earnings per share have risen from $1.50 to $2.97, while estimates for 2027 have increased from $2.91 to $4.81 during the same time. LQDA shares have surged 118.9% year to date.
Liquidia’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 54.40%.
View the Zacks #1 Rank List
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Regenxbio Shares Rise on Plans to Resubmit Navsunli BLA for MPS II
Key Takeaways
Regenxbio (RGNX - Free Report) announced that it has reached alignment with the FDA on the next regulatory steps for the potential accelerated approval of Navsunli (clemidsogene lanparvovec-sngl, RGX-121), a one-time investigational gene therapy being developed to treat patients with Mucopolysaccharidosis II (MPS II) or Hunter syndrome.
Shares of the company were up 16.3% yesterday following the announcement of the news.
Following recent constructive discussions, the FDA has now acknowledged that the existing clinical data are sufficient to support accelerated approval of Navsunli for MPS II, an ultra-rare neurodegenerative disease.
The regulatory body has asked the company to request a Type A meeting to review existing longer-term biomarker and clinical data, after which the company plans to resubmit the biologics license application (BLA) for Navsunli in the third quarter of 2026.
The Type A meeting is expected to be held in July next month.
RGNX’s Price Performance
Year to date, shares of Regenxbio have declined 37% compared with the industry’s decrease of 1.1%.
Image Source: Zacks Investment Research
RGNX’s Recent Developments With Navsunli
In February 2026, the FDA issued a complete response letter (CRL) to the BLA for Navsunli in MPS II.
Back then, the CRL outlined several possible paths forward, such as conducting a new study, collecting longer-term data from additional patients, or using an untreated control group.
Last month, the FDA lifted the clinical hold for Navsunli.
The FDA accepted the BLA for Navsunli under the accelerated approval pathway in May 2025.
Hunter syndrome is caused by a deficiency of the iduronate 2-sulfatase enzyme, leading to the buildup of harmful substances in tissues, including the brain, and resulting in progressive cognitive, motor and organ damage.
In March 2026, the FDA granted accelerated approval to Denali Therapeutics’ (DNLI - Free Report) tividenofusp alfa-eknm, under the brand name Avlayah, for the treatment of Hunter Syndrome. The continued approval for this indication may be contingent upon verification of clinical benefit in a confirmatory study.
Developed by Denali, Avlayah is enabled by its TransportVehicle platform, which facilitates delivery of biologics throughout the body, including the brain.
DNLI’s Avlayah is an enzyme replacement therapy indicated for pediatric patients with Hunter syndrome, targeting neurological symptoms when initiated early.
REGENXBIO Inc. Price
REGENXBIO Inc. price | REGENXBIO Inc. Quote
RGNX Zacks Rank & Stocks to Consider
Regenxbio currently carries a Zacks Rank #4 (Sell).
Some better-ranked stocks in the biotech sector are Immunocore (IMCR - Free Report) and Liquidia Corporation (LQDA - Free Report) , each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 60 days, estimates for Immunocore’s 2026 bottom line have improved from a loss of 88 cents per share to earnings of 6 cents. Over the same period, EPS estimates for 2027 have risen from 24 cents to 87 cents. IMCR stock has lost 15.4% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 46.66%.
Over the past 60 days, estimates for Liquidia’s 2026 earnings per share have risen from $1.50 to $2.97, while estimates for 2027 have increased from $2.91 to $4.81 during the same time. LQDA shares have surged 118.9% year to date.
Liquidia’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 54.40%.