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SNY's Rilzabrutinib Wins 4th Orphan Drug Tag for Sickle Cell Disease
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Key Takeaways
Sanofi's rilzabrutinib receives FDA orphan drug designation for SCD, its fourth such indication.
Preclinical data show rilzabrutinib reduced vaso-occlusion and inflammation in SCD mouse models.
Rilzabrutinib is also under FDA review for ITP, with a decision expected by August 29, 2025.
Sanofi (SNY - Free Report) announced that the FDA has granted orphan drug designation to its investigational BTK inhibitor, rilzabrutinib, for sickle cell disease (SCD). The candidate exerts its effects through multi-immune modulation, aiming to reduce vaso-occlusive crises, potentially by mitigating inflammation.
The latest FDA grant marks the fourth indication for which Sanofi’s rilzabrutinib enjoys the orphan drug designation. The FDA grants this designation to support the development of medicines for rare disorders that affect fewer than 200,000 patients in the United States.
The candidate, for the SCD indication, is yet to enter clinical-stage development. However, in preclinical studies, treatment with rilzabrutinib reduced both vaso-occlusion — blockage of blood vessels — and inflammation, in transgenic mice with SCD.
SCD is a rare genetic disorder where abnormally shaped red blood cells block blood flow, leading to severe pain and complications, such as infections, stroke, and organ damage. It affects more than 100,000 people in the United States, with patients typically facing a life expectancy about 20 years shorter than average.
Year to date, shares of Sanofi have gained 2% against the industry’s 3.2% decline.
Image Source: Zacks Investment Research
We remind the investors that in April 2025, the FDA granted orphan drug designation to rilzabrutinib for treating warm autoimmune hemolytic anemia (wAIHA) and IgG4-related disease (IgG4-RD), two rare diseases.
The candidate is being developed in separate mid-stage studies for treating wAIHA and IgG4-RD. Currently, there are no approved therapies for treating wAIHA and IgG4-RD. Both diseases have a significant unmet medical need.
Data from a phase IIb study evaluating rilzabrutinib for wAIHA showed that treatment with the candidate demonstrated clinically meaningful outcomes on response rate and disease markers. Sanofi’s rilzabrutinib also enjoys the orphan drug designation for the wAIHA indication in the EU.
Additionally, data from a phase IIa study evaluating rilzabrutinib in IgG4-RD patients showed that treatment with the candidate for 52 weeks led to a reduction in disease flare, other disease markers and glucocorticoid sparing.
SNY's Rilzabrutinib Under Review for Immune Thrombocytopenia
Sanofi has developed rilzabrutinib for treating immune thrombocytopenia (ITP), another rare, complex autoimmune disorder. The candidate enjoys the orphan drug designation for the ITP indication in the United States, the EU and Japan.
A regulatory application seeking approval for rilzabrutinib for treating ITP is currently under review in the United States. A final decision from the FDA is expected on Aug. 29, 2025. The regulatory body has previously granted a fast-track designation and an orphan drug designation to rilzabrutinib for treating ITP in the United States.
Besides the United States, rilzabrutinib is also under review in the EU and China as a potential treatment for ITP.
Rilzabrutinib was added to Sanofi’s pipeline with the acquisition of Principia Biopharma. The candidate is also being developed in mid-stage studies for other immune-mediated diseases like asthma and chronic spontaneous urticaria.
In the past 60 days, estimates for Bayer’s earnings per share have increased from $1.19 to $1.25 for 2025. During the same time, earnings per share have increased from $1.28 to $1.31 for 2026. Year to date, shares of Bayer have gained 45.9%.
BAYRY’s earnings beat estimates in one of the trailing four quarters, matched twice and missed on the remaining occasion, the average negative surprise being 13.91%.
In the past 60 days, estimates for Lexicon’s loss per share have narrowed from 37 cents to 32 cents for 2025. During the same time, loss per share estimates for 2026 have narrowed from 35 cents to 31 cents. Year to date, shares of LXRX have lost 14.4%.
LXRX’s earnings beat estimates in three of the trailing four quarters and missed the same on the remaining occasion, delivering an average surprise of 11.97%.
In the past 60 days, estimates for Amarin’s loss per share for 2025 have narrowed from $5.00 to $2.78. During the same time, loss per share estimates for 2026 have narrowed from $3.87 to $2.04. Year to date, shares of AMRN have gained 23.2%.
AMRN’s earnings beat estimates in two of the trailing four quarters, matched once and missed the same on the remaining occasion, delivering an average surprise of 29.11%.
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SNY's Rilzabrutinib Wins 4th Orphan Drug Tag for Sickle Cell Disease
Key Takeaways
Sanofi (SNY - Free Report) announced that the FDA has granted orphan drug designation to its investigational BTK inhibitor, rilzabrutinib, for sickle cell disease (SCD). The candidate exerts its effects through multi-immune modulation, aiming to reduce vaso-occlusive crises, potentially by mitigating inflammation.
The latest FDA grant marks the fourth indication for which Sanofi’s rilzabrutinib enjoys the orphan drug designation. The FDA grants this designation to support the development of medicines for rare disorders that affect fewer than 200,000 patients in the United States.
The candidate, for the SCD indication, is yet to enter clinical-stage development. However, in preclinical studies, treatment with rilzabrutinib reduced both vaso-occlusion — blockage of blood vessels — and inflammation, in transgenic mice with SCD.
SCD is a rare genetic disorder where abnormally shaped red blood cells block blood flow, leading to severe pain and complications, such as infections, stroke, and organ damage. It affects more than 100,000 people in the United States, with patients typically facing a life expectancy about 20 years shorter than average.
Year to date, shares of Sanofi have gained 2% against the industry’s 3.2% decline.
Image Source: Zacks Investment Research
We remind the investors that in April 2025, the FDA granted orphan drug designation to rilzabrutinib for treating warm autoimmune hemolytic anemia (wAIHA) and IgG4-related disease (IgG4-RD), two rare diseases.
The candidate is being developed in separate mid-stage studies for treating wAIHA and IgG4-RD. Currently, there are no approved therapies for treating wAIHA and IgG4-RD. Both diseases have a significant unmet medical need.
Data from a phase IIb study evaluating rilzabrutinib for wAIHA showed that treatment with the candidate demonstrated clinically meaningful outcomes on response rate and disease markers. Sanofi’s rilzabrutinib also enjoys the orphan drug designation for the wAIHA indication in the EU.
Additionally, data from a phase IIa study evaluating rilzabrutinib in IgG4-RD patients showed that treatment with the candidate for 52 weeks led to a reduction in disease flare, other disease markers and glucocorticoid sparing.
SNY's Rilzabrutinib Under Review for Immune Thrombocytopenia
Sanofi has developed rilzabrutinib for treating immune thrombocytopenia (ITP), another rare, complex autoimmune disorder. The candidate enjoys the orphan drug designation for the ITP indication in the United States, the EU and Japan.
A regulatory application seeking approval for rilzabrutinib for treating ITP is currently under review in the United States. A final decision from the FDA is expected on Aug. 29, 2025. The regulatory body has previously granted a fast-track designation and an orphan drug designation to rilzabrutinib for treating ITP in the United States.
Besides the United States, rilzabrutinib is also under review in the EU and China as a potential treatment for ITP.
Rilzabrutinib was added to Sanofi’s pipeline with the acquisition of Principia Biopharma. The candidate is also being developed in mid-stage studies for other immune-mediated diseases like asthma and chronic spontaneous urticaria.
Sanofi Price and Consensus
Sanofi price-consensus-chart | Sanofi Quote
SNY's Zacks Rank & Other Stocks to Consider
Sanofi currently carries a Zacks Rank #2 (Buy).
Some other top-ranked stocks in the biotech sector are Bayer (BAYRY - Free Report) , Lexicon Pharmaceuticals (LXRX - Free Report) and Amarin (AMRN - Free Report) , each carrying a Zacks Rank #2 at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
In the past 60 days, estimates for Bayer’s earnings per share have increased from $1.19 to $1.25 for 2025. During the same time, earnings per share have increased from $1.28 to $1.31 for 2026. Year to date, shares of Bayer have gained 45.9%.
BAYRY’s earnings beat estimates in one of the trailing four quarters, matched twice and missed on the remaining occasion, the average negative surprise being 13.91%.
In the past 60 days, estimates for Lexicon’s loss per share have narrowed from 37 cents to 32 cents for 2025. During the same time, loss per share estimates for 2026 have narrowed from 35 cents to 31 cents. Year to date, shares of LXRX have lost 14.4%.
LXRX’s earnings beat estimates in three of the trailing four quarters and missed the same on the remaining occasion, delivering an average surprise of 11.97%.
In the past 60 days, estimates for Amarin’s loss per share for 2025 have narrowed from $5.00 to $2.78. During the same time, loss per share estimates for 2026 have narrowed from $3.87 to $2.04. Year to date, shares of AMRN have gained 23.2%.
AMRN’s earnings beat estimates in two of the trailing four quarters, matched once and missed the same on the remaining occasion, delivering an average surprise of 29.11%.