Sanofi’s rilzabrutinib designated breakthrough therapy in the US and orphan drug in Japan for the treatment of warm autoimmune hemolytic anemia
•Rilzabrutinib is the first and only investigational BTKi for warm autoimmune hemolytic anemia to be designated Breakthrough Therapy by the FDA
•Rilzabrutinib helps address complex immune-system dysregulation through multi-immune modulation
Rilzabrutinib holds global regulatory designations across multiple rare diseases, underscoring its broad therapeutic potential
The US Food and Drug Administration (FDA) has granted a designation as breakthrough therapy to Wayrilz (rilzabrutinib), a novel oral, reversible Bruton’s tyrosine kinase (BTK) inhibitor, for the treatment of patients with warm autoimmune hemolytic anemia (wAIHA), a rare autoimmune disorder marked by the destruction of red blood cells. The Japanese Ministry of Health, Labour and Welfare has also provided rilzabrutinib an orphan designation for the same condition.
Both designations are based on clinical data from the ongoing LUMINA 2 phase 2b study (clinical study identifier: NCT05002777) assessing the efficacy and safety of rilzabrutinib for patients with wAIHA. In addition, the new LUMINA 3 phase 3 study (clinical study identifier: NCT07086976), is assessing rilzabrutinib compared with placebo in patients with wAIHA. There is currently no approved treatment that specifically targets the underlying cause of this rare autoimmune condition, which can lead to anemia, fatigue, and serious organ damage.
An FDA breakthrough therapy designation is designed to expedite the development and review of medicines in the US intended to treat serious or life-threatening conditions and where preliminary clinical evidence indicates the therapy may demonstrate substantial improvement over available treatment options. Orphan designation in Japan is granted to medicines intended to address rare diseases with high unmet medical need.
“These recognitions highlight the critical unmet need that persists for people living with wAIHA,” said Karin Knobe, Global Head of Development, Rare Diseases. “Furthermore, receiving such designations reinforces our commitment to advancing innovative medicines for rare diseases that currently have limited or no approved treatment options.”
Rilzabrutinib is approved in the US, the EU, and the United Arab Emirates (UAE) under the brand name Wayrilz for the treatment of adults with immune thrombocytopenia (ITP) and is currently under regulatory review for ITP in Japan. Other than the approved ITP indications in the US, EU, and UAE, these uses of rilzabrutinib are investigational and have not been evaluated by any regulatory authority.
