Sanofi has received approval from the FDA for its oral drug, Wayrilz (rilzabrutinib), the first Bruton’s tyrosine kinase (BTK) inhibitor for persistent or chronic immune thrombocytopenia (ITP).
ITP is a complex immune dysregulation disease that causes low platelet counts and bleeding. Wayrilz is a novel, reversible BTK inhibitor that addresses the root causes of ITP through multi-immune modulation by targeting various immune system pathways.
Wayrilz's approval was based on positive results in the LUNA phase 3 study, where the treatment led to improvements in ITP symptoms, including a sustainable platelet response that was statistically significant at week 25 in 23% of patients (compared to 0% with placebo). Patients taking Wayrilz also had a faster time to first platelet response and a longer duration of platelet response than those taking the placebo.
Wayrilz will be available for patients who have had an insufficient response to an earlier treatment for ITP. It is expected that more than 25,000 adults in the U.S. could benefit from such treatment. The drug is already approved in the United Arab Emirates and is under regulatory review in the EU and China.