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Sanofi's Venglustat Wins FDA Breakthrough Tag for Gaucher Disease
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Key Takeaways
Sanofi's venglustat wins FDA Breakthrough Therapy tag for type III Gaucher disease.
Phase III LEAP2MONO study showed significant neurological improvement vs Cerezyme at 52 weeks.
Sanofi plans global regulatory filings for venglustat in Gaucher disease in 2026 after strong study results.
Sanofi (SNY - Free Report) announced that the FDA has granted Breakthrough Therapy designation to venglustat, an investigational oral glucosylceramide synthase inhibitor, for the treatment of patients with type III Gaucher disease (GD3).
The FDA’s Breakthrough Therapy designation is intended to facilitate drug development and expedite the review of drugs for serious or life-threatening diseases when early clinical data suggest meaningful advantages over existing treatments.
Over the past year, SNY’s shares have plunged 24.8% against the industry’s 8.2% growth.
Image Source: Zacks Investment Research
FDA’s Breakthrough Tag Supported by Late-Stage Study
The Breakthrough Therapy designation for venglustat is based on data from the phase III LEAP2MONO study, which evaluated the safety and efficacy of once-daily venglustat against Cerezyme (imiglucerase), an intravenous enzyme replacement therapy marketed by Sanofi. This study enrolled patients aged 12 years and older with GD3 who were previously stable on ERT.
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.
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.
Following the successful phase III data, Sanofi anticipates global regulatory filings for venglustat to treat GD3 in 2026.
More on Sanofi’s Venglustat
The FDA previously granted 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.
SNY’s Zacks Rank & Stocks to Consider
SNY currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Catalyst Pharmaceuticals and Indivior Pharmaceuticals (INDV - Free Report) , each currently sporting a Zacks Rank #1 (Strong Buy), and ANI Pharmaceuticals (ANIP - Free Report) , which presently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 60 days, estimates for Catalyst Pharmaceuticals’ 2026 earnings per share have risen from $2.55 to $2.82. CPRX shares have gained 3.2% over the past year.
Catalyst Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 35.19%.
Over the past 60 days, estimates for Indivior Pharmaceuticals’ 2026 earnings per share have risen from $2.79 to $3.03. INDV shares have surged 214.9% over the past year.
Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 74.53%.
Over the past 60 days, estimates for ANI Pharmaceuticals’ earnings per share have increased from $8.28 to $9.00 for 2026. Over the past year, shares of ANIP have rallied 14.5%.
ANI Pharmaceuticals' earnings beat estimates in each of the trailing four quarters, with the average surprise being 22.21%.
View the Zacks #1 Rank List
Image: Bigstock
Sanofi's Venglustat Wins FDA Breakthrough Tag for Gaucher Disease
Key Takeaways
Sanofi (SNY - Free Report) announced that the FDA has granted Breakthrough Therapy designation to venglustat, an investigational oral glucosylceramide synthase inhibitor, for the treatment of patients with type III Gaucher disease (GD3).
The FDA’s Breakthrough Therapy designation is intended to facilitate drug development and expedite the review of drugs for serious or life-threatening diseases when early clinical data suggest meaningful advantages over existing treatments.
Over the past year, SNY’s shares have plunged 24.8% against the industry’s 8.2% growth.
Image Source: Zacks Investment Research
FDA’s Breakthrough Tag Supported by Late-Stage Study
The Breakthrough Therapy designation for venglustat is based on data from the phase III LEAP2MONO study, which evaluated the safety and efficacy of once-daily venglustat against Cerezyme (imiglucerase), an intravenous enzyme replacement therapy marketed by Sanofi. This study enrolled patients aged 12 years and older with GD3 who were previously stable on ERT.
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.
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.
Following the successful phase III data, Sanofi anticipates global regulatory filings for venglustat to treat GD3 in 2026.
More on Sanofi’s Venglustat
The FDA previously granted 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.
SNY’s Zacks Rank & Stocks to Consider
SNY currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Catalyst Pharmaceuticals and Indivior Pharmaceuticals (INDV - Free Report) , each currently sporting a Zacks Rank #1 (Strong Buy), and ANI Pharmaceuticals (ANIP - Free Report) , which presently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 60 days, estimates for Catalyst Pharmaceuticals’ 2026 earnings per share have risen from $2.55 to $2.82. CPRX shares have gained 3.2% over the past year.
Catalyst Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 35.19%.
Over the past 60 days, estimates for Indivior Pharmaceuticals’ 2026 earnings per share have risen from $2.79 to $3.03. INDV shares have surged 214.9% over the past year.
Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 74.53%.
Over the past 60 days, estimates for ANI Pharmaceuticals’ earnings per share have increased from $8.28 to $9.00 for 2026. Over the past year, shares of ANIP have rallied 14.5%.
ANI Pharmaceuticals' earnings beat estimates in each of the trailing four quarters, with the average surprise being 22.21%.