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SNY's Rare Disease Drug Wins FDA Breakthrough and Japan Orphan Status
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Key Takeaways
The FDA granted Breakthrough Therapy to Wayrilz for wAIHA, and Japan awarded Orphan Drug status.
Sanofi's designations are supported by LUMINA 2 data, with the late-stage LUMINA 3 study underway.
Wayrilz is approved for chronic ITP in the U.S., EU and UAE, and is under review in Japan.
Sanofi (SNY - Free Report) announced that the FDA has granted Breakthrough Therapy designation to Wayrilz (rilzabrutinib), a novel oral, reversible Bruton’s tyrosine kinase (BTK) inhibitor, for the treatment of patients with warm autoimmune hemolytic anemia (wAIHA). Concurrently, Japan’s Ministry of Health, Labour and Welfare granted Orphan Drug designation to the drug for the same indication.
Both regulatory designations are supported by clinical data from the ongoing LUMINA 2 phase IIb study evaluating the efficacy and safety of rilzabrutinib in patients with wAIHA. In addition, the late-stage LUMINA 3 study is underway to assess rilzabrutinib versus placebo in this patient population.
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 suggests meaningful advantages over existing treatments.
Over the past year, SNY’s shares have declined 10.5% against the industry’s 17.1% rise.
Image Source: Zacks Investment Research
Rilzabrutinib is already approved under the brand name Wayrilz in the United States, the European Union and the United Arab Emirates for the treatment of persistent or chronic immune thrombocytopenia (ITP) in adult patients who have had an insufficient response to a previous treatment. With this, Wayrilz becomes the first BTK inhibitor approved for ITP. Wayrilz is currently under regulatory review in Japan.
Wayrilz was added to Sanofi’s pipeline through the acquisition of Principia Biopharma in 2020.
Key Updates on Sanofi’s Wayrilz for Autoimmune Disorders
Beyond wAIHA and ITP, Sanofi continues to advance Wayrilz across several other rare autoimmune diseases globally.
Sanofi is evaluating Wayrilz for the treatment of patients with IgG4-related disease (IgG4-RD), a chronic, immune-mediated rare condition that can affect multiple organs, causing inflammation, swelling and fibrosis.
The FDA previously granted Orphan Drug designation to Wayrilz for autoimmune hemolytic anemia, IgG4-RD and sickle cell disease, along with Fast Track designation for ITP and IgG4-RD, while the European Union awarded Orphan Drug designation for ITP, autoimmune hemolytic anemia and IgG4-RD.
wAIHA is a rare, potentially life-threatening autoimmune disorder marked by immune-mediated red blood cell destruction that can result in significant anemia, fatigue and potentially serious organ complications. It accounts for over half of all autoimmune hemolytic anemia cases. At present, there are no approved therapies for this indication.
Over the past 60 days, estimates for Assertio’s 2026 loss per share have narrowed from 30 cents to 28 cents. ASRT shares have lost 2.3% over the past year.
Assertio’s earnings beat estimates in one quarter and missed in the remaining three quarters, with the average negative surprise being 35.21%.
Over the past 60 days, estimates for Alkermes’ 2026 earnings per share have increased from $1.54 to $1.91. ALKS shares have risen 11.6% over the past year.
Alkermes’ earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average earnings surprise being 4.58%.
Over the past 60 days, estimates for Castle Biosciences’ 2026 loss per share have narrowed from $1.06 to 96 cents. CSTL shares have risen 16.3% over the past year.
Castle Biosciences’ earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 66.11%.
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SNY's Rare Disease Drug Wins FDA Breakthrough and Japan Orphan Status
Key Takeaways
Sanofi (SNY - Free Report) announced that the FDA has granted Breakthrough Therapy designation to Wayrilz (rilzabrutinib), a novel oral, reversible Bruton’s tyrosine kinase (BTK) inhibitor, for the treatment of patients with warm autoimmune hemolytic anemia (wAIHA). Concurrently, Japan’s Ministry of Health, Labour and Welfare granted Orphan Drug designation to the drug for the same indication.
Both regulatory designations are supported by clinical data from the ongoing LUMINA 2 phase IIb study evaluating the efficacy and safety of rilzabrutinib in patients with wAIHA. In addition, the late-stage LUMINA 3 study is underway to assess rilzabrutinib versus placebo in this patient population.
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 suggests meaningful advantages over existing treatments.
Over the past year, SNY’s shares have declined 10.5% against the industry’s 17.1% rise.
Image Source: Zacks Investment Research
Rilzabrutinib is already approved under the brand name Wayrilz in the United States, the European Union and the United Arab Emirates for the treatment of persistent or chronic immune thrombocytopenia (ITP) in adult patients who have had an insufficient response to a previous treatment. With this, Wayrilz becomes the first BTK inhibitor approved for ITP. Wayrilz is currently under regulatory review in Japan.
Wayrilz was added to Sanofi’s pipeline through the acquisition of Principia Biopharma in 2020.
Key Updates on Sanofi’s Wayrilz for Autoimmune Disorders
Beyond wAIHA and ITP, Sanofi continues to advance Wayrilz across several other rare autoimmune diseases globally.
Sanofi is evaluating Wayrilz for the treatment of patients with IgG4-related disease (IgG4-RD), a chronic, immune-mediated rare condition that can affect multiple organs, causing inflammation, swelling and fibrosis.
The FDA previously granted Orphan Drug designation to Wayrilz for autoimmune hemolytic anemia, IgG4-RD and sickle cell disease, along with Fast Track designation for ITP and IgG4-RD, while the European Union awarded Orphan Drug designation for ITP, autoimmune hemolytic anemia and IgG4-RD.
wAIHA is a rare, potentially life-threatening autoimmune disorder marked by immune-mediated red blood cell destruction that can result in significant anemia, fatigue and potentially serious organ complications. It accounts for over half of all autoimmune hemolytic anemia cases. At present, there are no approved therapies for this indication.
SNY’s Zacks Rank & Stocks to Consider
SNY currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Assertio Holdings (ASRT - Free Report) , Alkermes (ALKS - Free Report) and Castle Biosciences (CSTL - 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 Assertio’s 2026 loss per share have narrowed from 30 cents to 28 cents. ASRT shares have lost 2.3% over the past year.
Assertio’s earnings beat estimates in one quarter and missed in the remaining three quarters, with the average negative surprise being 35.21%.
Over the past 60 days, estimates for Alkermes’ 2026 earnings per share have increased from $1.54 to $1.91. ALKS shares have risen 11.6% over the past year.
Alkermes’ earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average earnings surprise being 4.58%.
Over the past 60 days, estimates for Castle Biosciences’ 2026 loss per share have narrowed from $1.06 to 96 cents. CSTL shares have risen 16.3% over the past year.
Castle Biosciences’ earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 66.11%.