Amit Mehta, MD, Medical Oncologist, discusses data that led to the approval of Wayrilz (rilzabrutinib) for adult patients with immune thrombocytopenia (ITP).
ITP is a bleeding disorder characterized by low levels of platelets. It is an autoimmune disorder in which abnormal antibodies attach to the platelet membranes. 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.
The U.S. Food and Drug Administration (FDA) recently approved rilzabrutinib for adults with persistent or chronic ITP who have had an insufficient response to previous treatment. The approval was based on data from the LUNA 3 clinical trial (NCT04562766), a phase 3, randomized, multicenter study evaluating the safety and efficacy of rilzabrutinib versus placebo in adult and adolescent patients with persistent or chronic ITP. Results from the adult group of 202 patients were presented at the 66th American Society of Hematology Annual Meeting and Exposition. The adolescent part is ongoing.
Rilzabrutinib is an oral, reversible, Bruton’s tyrosine kinase (BTK) inhibitor that addresses the underlying cause of ITP through multi-immune modulation and targeting various pathways across the immune system.
LUNA 3 Trial Results
Patients who achieved platelet count response at 12 weeks were eligible to continue the full 24-week double-blind period. This included 64% of patients in the rilzabrutinib arm and 32% of patients in the placebo arm.
In the rilzabrutinib arm, 23% of patients experienced statistically significant durable platelet response at week 25 compared to 0% in the placebo arm. Rilzabrutinib patients also experienced a faster time to first platelet response of 36 days compared to the platelet response not being reached in the placebo group. The treatment group also showed a longer duration of platelet response of 7 weeks compared to 0.7 weeks in the placebo group.
Patients in the rilzabrutinib arm also reported an overall 10.6-point improvement in nine health-related quality of life measures, compared to a 2.3-point increase in the placebo group.
The most common adverse reactions are diarrhea, nausea, headache, abdominal pain, and COVID-19.
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To learn more about ITP and other rare hematologic conditions, visit https://checkrare.com/diseases/hematologic-disorders/