Sanofi Received Breakthrough Therapy and Orphan Drug Designations for Rilzabrutinib
Sanofi has announced that Rilzabrutinib has received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for the treatment of warm autoimmune hemolytic anemia (wAIHA).
The Japanese Ministry of Health, Labour and Welfare has also awarded the medicine orphan drug designation for the same condition.
These regulatory recognitions are based on data from the ongoing LUMINA 2 phase 2b study evaluating the safety and efficacy of rilzabrutinib in patients with wAIHA.
A phase 3 trial, LUMINA 3, is further assessing the drug versus placebo. Currently, no approved therapies directly target the underlying cause of wAIHA, a rare autoimmune disorder that leads to premature destruction of red blood cells and may cause fatigue, dizziness, shortness of breath, and serious complications such as blood clots.
Rilzabrutinib, marketed as Wayrilz, is approved in the US, EU, and UAE for the treatment of adults with immune thrombocytopenia (ITP) and is under regulatory review for ITP in Japan.
The oral, reversible covalent Bruton’s tyrosine kinase (BTK) inhibitor works by modulating immune pathways and restoring immune balance.
In addition to ITP and wAIHA, rilzabrutinib is being investigated for other rare immune-mediated conditions, including IgG4-related disease and sickle cell disease. These additional uses remain under study and are not yet approved.
