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FDA Grants Priority Review to SNY's Filing for Gaucher Disease Drug
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Key Takeaways
SNY's venglustat won FDA priority review for GD3, with a target action date of Nov. 25, 2026.
Phase III LEAP2MONO study showed significant neurological symptom improvement at 52 weeks.
Sanofi's venglustat previously received Breakthrough Therapy, Fast-Track and Orphan Drug tags.
Sanofi (SNY - Free Report) announced that the FDA has accepted its new drug application (NDA) seeking approval for venglustat, an investigational oral glucosylceramide synthase inhibitor, for the treatment of patients with type III Gaucher disease (GD3).
The FDA has granted a priority review to the filing and a final decision is expected by Nov. 25, 2026. If approved, venglustat would become the first therapy in the United States to address the progressive neurological symptoms of GD3.
A Priority Review designation means the FDA’s goal is to take action on an application within six months compared with 10 months under standard review.
Venglustat is currently under regulatory review in the European Union for GD3. SNY plans to submit additional global filings for GD3 in 2026.
Gaucher disease is a rare genetic lysosomal disorder caused by a deficiency of the enzyme glucocerebrosidase, which leads to the buildup of glycosphingolipids in cells, especially in macrophages within the spleen, liver, bone marrow and lungs.
Year to date, SNY’s shares have declined 8.6% against the industry’s 2.3% growth.
Image Source: Zacks Investment Research
Late-Stage Study Supports SNY’s Venglustat NDA
The NDA filing is based on positive data from the phase III LEAP2MONO study, which evaluated venglustat in patients aged 12 years and older with GD3 who were stable on enzyme replacement therapy (ERT) against Cerezyme (imiglucerase), an intravenous ERT marketed by Sanofi.
The study met all its primary endpoints, demonstrating statistically significant and clinically meaningful improvements in neurological symptoms at 52 weeks compared with patients receiving ERT.
Cerezyme is currently approved to treat the non-central nervous system symptoms of type I Gaucher disease or GD3 in adults and pediatric patients. The approval in the GD3 indication was received earlier this year in January.
Sanofi is evaluating the effect of venglustat on left cardiac ventricular mass index in men and women with Fabry disease, another rare lysosomal storage disorder in an ongoing late-stage CARAT study.
The FDA previously granted Breakthrough Therapy and Fast-Track designation to Sanofi’s venglustat for its potential use in GD3. The candidate was also granted Orphan Drug designation for GD3 in the United States, the European Union and Japan.
Marketed Drugs for Gaucher Disease
SNY’s commercial portfolio for the treatment of Gaucher disease includes two globally marketed drugs, Cerezyme and Cerdelga, which provide enzyme replacement and oral treatment options, respectively.
Over the past 30 days, estimates for Indivior Pharmaceuticals’ 2026 earnings per share have increased from $3.00 to $3.35. Over the same period, EPS estimates for 2027 have risen to $3.33 from $3.69. INDV shares have risen 2% year to date.
Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 65.44%.
Over the past 30 days, estimates for Liquidia’s 2026 earnings per share have increased to $2.97 from $1.50. Over the same period, EPS estimates for 2027 have risen to $4.81 from $2.91. LQDA shares have gained 79.9% year to date.
Liquidia’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 54.40%.
Over the past 30 days, estimates for Immunocore’s 2026 loss per share have narrowed from 97 cents to 16 cents. Over the same period, estimates for 2027 have improved from a loss of 39 cents to earnings of 11 cents per share. IMCR shares have lost 16.5% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 46.66%.
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FDA Grants Priority Review to SNY's Filing for Gaucher Disease Drug
Key Takeaways
Sanofi (SNY - Free Report) announced that the FDA has accepted its new drug application (NDA) seeking approval for venglustat, an investigational oral glucosylceramide synthase inhibitor, for the treatment of patients with type III Gaucher disease (GD3).
The FDA has granted a priority review to the filing and a final decision is expected by Nov. 25, 2026. If approved, venglustat would become the first therapy in the United States to address the progressive neurological symptoms of GD3.
A Priority Review designation means the FDA’s goal is to take action on an application within six months compared with 10 months under standard review.
Venglustat is currently under regulatory review in the European Union for GD3. SNY plans to submit additional global filings for GD3 in 2026.
Gaucher disease is a rare genetic lysosomal disorder caused by a deficiency of the enzyme glucocerebrosidase, which leads to the buildup of glycosphingolipids in cells, especially in macrophages within the spleen, liver, bone marrow and lungs.
Year to date, SNY’s shares have declined 8.6% against the industry’s 2.3% growth.
Image Source: Zacks Investment Research
Late-Stage Study Supports SNY’s Venglustat NDA
The NDA filing is based on positive data from the phase III LEAP2MONO study, which evaluated venglustat in patients aged 12 years and older with GD3 who were stable on enzyme replacement therapy (ERT) against Cerezyme (imiglucerase), an intravenous ERT marketed by Sanofi.
The study met all its primary endpoints, demonstrating statistically significant and clinically meaningful improvements in neurological symptoms at 52 weeks compared with patients receiving ERT.
Cerezyme is currently approved to treat the non-central nervous system symptoms of type I Gaucher disease or GD3 in adults and pediatric patients. The approval in the GD3 indication was received earlier this year in January.
Sanofi is evaluating the effect of venglustat on left cardiac ventricular mass index in men and women with Fabry disease, another rare lysosomal storage disorder in an ongoing late-stage CARAT study.
The FDA previously granted Breakthrough Therapy and Fast-Track designation to Sanofi’s venglustat for its potential use in GD3. The candidate was also granted Orphan Drug designation for GD3 in the United States, the European Union and Japan.
Marketed Drugs for Gaucher Disease
SNY’s commercial portfolio for the treatment of Gaucher disease includes two globally marketed drugs, Cerezyme and Cerdelga, which provide enzyme replacement and oral treatment options, respectively.
SNY’s Zacks Rank & Stocks to Consider
SNY currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Indivior Pharmaceuticals (INDV - Free Report) , Liquidia Corporation (LQDA - Free Report) , and Immunocore (IMCR - 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 30 days, estimates for Indivior Pharmaceuticals’ 2026 earnings per share have increased from $3.00 to $3.35. Over the same period, EPS estimates for 2027 have risen to $3.33 from $3.69. INDV shares have risen 2% year to date.
Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 65.44%.
Over the past 30 days, estimates for Liquidia’s 2026 earnings per share have increased to $2.97 from $1.50. Over the same period, EPS estimates for 2027 have risen to $4.81 from $2.91. LQDA shares have gained 79.9% year to date.
Liquidia’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 54.40%.
Over the past 30 days, estimates for Immunocore’s 2026 loss per share have narrowed from 97 cents to 16 cents. Over the same period, estimates for 2027 have improved from a loss of 39 cents to earnings of 11 cents per share. IMCR shares have lost 16.5% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 46.66%.