RARE Rises on Completion of Rolling Submission for AAV Gene Therapy

Shares of Ultragenyx Pharmaceutical RARE were up 15.5% yesterday following the completion of the rolling submission of a biologics license application (“BLA”) to the FDA seeking approval for its AAV gene therapy, DTX401 (pariglasgene brecaparvovec), for the treatment of glycogen storage disease type Ia (GSDIa).

GSDIa is a rare, life-threatening metabolic disorder that leads to severe hypoglycemia (excess glycogen storage) and other complications. Managing the disease relies on frequent cornstarch intake, but current approaches are burdensome, and no approved pharmacologic treatments exist. GSDIa affects around 6,000 people in commercially accessible regions.

If approved, DTX401 will become the first therapy to be approved for treating the underlying cause of GSDIa.

The BLA was supported by data from a robust clinical program. Previously, results from the phase III GlucoGene study showed that treatment with DTX401 led to significant and clinically meaningful reductions in both the quantity and frequency of daily cornstarch use while maintaining low hypoglycemia rates, improving blood glucose control and increasing fasting tolerance. Treatment with DTX401 was also well tolerated with an acceptable safety profile.

RARE Price Performance

In the past six months, shares of Ultragenyx have lost 37.2% against the industry’s rise of 25.2%.

RARE’s Ongoing Development Activities With DTX401

In August 2025, RARE initiated the rolling submission of a BLA to the FDA for DTX401 in GSDIa and submitted the non-clinical and clinical modules. The company has now completed the package by submitting the chemistry, manufacturing, and controls module.

In September 2025, Ultragenyx announced positive longer-term datafrom a late-stage study of DTX401 for the treatment of GSDIa. Per the company, GSDIa patients achieved greater reductions in daily cornstarch intake while sustaining low hypoglycemia, enhanced euglycemia and better fasting tolerance following 96 weeks of treatment with the candidate.

Last year, Ultragenyx reported achieving the primary endpoint in the same phase III GSDIa study. Per these results, treatment with DTX401 led to a statistically significant and clinically meaningful reduction in daily cornstarch intake of 41% at week 48 compared with a 10% reduction in the placebo group.

The completion of the rolling submission of the BLA for DTX401 in GSDIa marks a significant step forward for Ultragenyx in delivering a potential first-in-class therapy for this rare metabolic disorder.

Ultragenyx Pharmaceutical Inc. Price

Ultragenyx Pharmaceutical Inc. price | Ultragenyx Pharmaceutical Inc. Quote

RARE’s Zacks Rank & Stocks to Consider

Ultragenyx currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the biotech sector are CorMedix CRMD, ANI Pharmaceuticals ANIP and Castle Biosciences CSTL, each sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, estimates for CorMedix’s 2025 earnings per share (EPS) have increased from $1.85 to $2.87. EPS estimates for 2026 have moved up from $2.49 to $2.88 during the same period. CRMD stock has increased 5.9% in the past six months.

CorMedix’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 27.04%.

In the past 60 days, estimates for ANI Pharmaceuticals’ 2025 EPS have increased from $7.29 to $7.56. EPS estimates for 2026 have moved up from $7.81 to $8.08 during the same period. ANIP stock has rallied 24.9% in the past six months.

ANI Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 21.24%.

In the past 60 days, estimates for Castle Biosciences’ loss per share have narrowed from 64 cents to 34 cents for 2025. During the same time, loss per share estimates for 2026 have narrowed from $1.82 to $1.06. In the past six months, shares of CSTL have rallied 93.2%.

Castle Biosciences’ earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, the average surprise being 66.11%.

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This article originally published on Zacks Investment Research (zacks.com).

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