The U.S. Food and Drug Administration (FDA) has approved Wayrilz (rilzabrutinib) for the treatment of adults with persistent or chronic immune thrombocytopenia (ITP) who have had insufficient response to previous treatment.
ITP is a rare bleeding disorder characterized by too few platelets in the blood. This is because platelets are being destroyed by the immune system. Symptoms may include bruising, nosebleed or bleeding in the mouth, bleeding into the skin, and abnormally heavy menstruation. Rarely, ITP may become a chronic ailment in adults and reappear, even after remission.
Rilzabrutinib is the first Bruton’s tyrosine kinase (BTK) inhibitor for ITP that targets the root cause of disease through multi-immune modulation. The approval follows data from the phase 3, randomized, multicenter, LUNA 3 clinical trial evaluating the safety and efficacy of rilzabrutinib versus placebo in patients with persistent or chronic ITP.
Data from the study was presented at the 66th American Society of Hematology Annual Meeting and Exposition. Patients receiving rilzabrutinib experienced statistically significant durable platelet response of 23% at week 25, compared to 0% in the placebo group. Patients on treatment also experienced faster time to first platelet response and longer duration of platelet response compared to placebo. These results were consistent with primary and key secondary endpoints of the study.
Additionally, patients on rilzabrutinib reported an overall 10.6 point improvement across nine health-related quality of life measures, compared to a 2.3 point improvement in the placebo group. The most common adverse events were diarrhea, nausea, headache, abdominal pain, and COVID-19.
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To learn more about ITP and other rare hematologic disorders, visit https://checkrare.com/diseases/hematologic-disorders/
