Sanofi (SNY) announced that the US Food and Drug Administration has granted a breakthrough therapy designation to Wayrilz or Rilzabrutinib for the treatment of patients with warm autoimmune haemolytic anaemia or wAIHA, a rare autoimmune disorder marked by the destruction of red blood cells.
Separately, the Japanese Ministry of Health, Labour and Welfare has granted orphan drug designation to Rilzabrutinib for the same condition.
Both designations are based on clinical data from the ongoing LUMINA 2 phase 2b study assessing the efficacy and safety of Rilzabrutinib for patients with wAIHA.
Rilzabrutinib is an oral, reversible, covalent inhibitor of Bruton's tyrosine kinase.
The phase 2 study analysing the impact of Rilzabrutinib on biomarkers and supplement factors in wAIHA observed changes in biomarkers and complement activity, suggesting that Rilzabrutinib may reduce neutrophil activation, adhesion, inflammation, and complement activation.
Rilzabrutinib is approved in the US, the EU, and the United Arab Emirates under the brand name Wayrilz for the treatment of adults with immune thrombocytopenia or ITP and is currently under regulatory review for ITP in Japan.
In addition to ITP and wAIHA, Rilzabrutinib is being studied across a variety of rare diseases, including IgG4-RD and SCD.
The FDA had previously granted orphan drug designation to Rilzabrutinib for autoimmune haemolytic anaemia, as well as two other rare diseases: IgG4-related disease or IgG4-RD and sickle cell disease.
Rilzabrutinib also received FDA fast-track designation for ITP and IgG4-RD, and EU orphan designation for ITP, autoimmune haemolytic anaemia, and IgG4-related disease (IgG4-RD).
"These recognitions highlight the critical unmet need that persists for people living with wAIHA," said Karin Knobe, Global Head of Development, Rare Diseases.
SNY has traded between $44.62 and $60.12 in the last year.The stock closed Friday's trade at $47.83, up 0.72%.
In the overnight trading, SNY is up 0.65% at $47.52.
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