Today, Sanofi announced that its investigational oral BTK inhibitor, Rilzabrutinib, has been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) and Orphan Drug designation by the Japanese Ministry of Health, Labour and Welfare (MHLW) for the treatment of warm autoimmune hemolytic anemia (wAIHA).According to Sanofi's announcement, both recognitions are based on clinical data from the ongoing LUMINA Phase 2b study (Clinical Study No.: NCT05002777), which aims to evaluate the efficacy and safety of rizabutinib in patients with wAIHA. In addition, the newly initiated LUMINA Phase 3 study (Clinical Study No.: NCT07086976) is evaluating the efficacy of rizabutinib versus placebo in patients with wAIHA.In addition, the drug has been approved for marketing in the United States, the European Union and the United Arab Emirates (UAE) under the brand name Wayrilz for the treatment of immune thrombocytopenic purpura (ITP) in adults. (Image source: Sanofi official website)
Follow NewDrugs.com for daily updates on new drugs from around the world.

wAIHA is a rare and serious autoimmune blood disorder in which patients produce antibodies against their own red blood cells, leading to premature destruction of these cells and causing symptoms such as anemia, fatigue, and jaundice. In severe cases, it can be life-threatening. Currently, treatment options for this disease are limited, and some patients do not respond well to or tolerate existing therapies poorly, highlighting the urgent need for innovative treatment options.

Rilzabrutinib is an oral, reversible Bruton's tyrosine kinase (BTK) inhibitor designed to reduce autoantibody-mediated erythrocyte destruction by modulating B cells and innate immune-related pathways.

The original developer of this drug was Principia Biopharma Inc., which was later acquired by Sanofi, which then advanced its development and global research efforts.

Previously, the drug had received orphan drug designation from the FDA for the treatment of autoimmune hemolytic anemia, IgG4-related disease (IgG4-RD), and sickle cell disease (SCD); and from the EU for ITP, autoimmune hemolytic anemia, and IgG4-RD.

In China, Rilzabrutinib was submitted for import approval in December 2024 ( presumably for ITP ). Currently, supplementary data is being collected, but the deadline for submitting supplementary data is approaching, and a final approval result is expected in March or April 2026. In addition, Rilzabrutinib has also received approval for three clinical trials in China.

October 2025, IgG4-related diseases;

In July 2025, warm antibody-type autoimmune hemolytic anemia (wAIHA).

In January 2025, adults and adolescents aged 16 years and older will be classified as having primary focal segmental glomerulosclerosis (FSGS) or primary minimal change disease (MCD).

https://bydrug.pharmcube.com/news/detail/5bb801884b8a78acdf5e240572604e27