The U.S. Food and Drug Administration (FDA) has approved Wayrilz (rilzabrutinib), an oral Bruton’s tyrosine kinase (BTK) inhibitor, for adults with persistent or chronic immune thrombocytopenia (ITP) who have not adequately responded to previous treatments. This approval marks the first BTK inhibitor authorized for this condition.
The decision was based on results from the pivotal Phase 3 LUNA 3 study, which demonstrated that Wayrilz significantly improved sustained platelet counts and other ITP symptoms compared to placebo. Patients treated with Wayrilz showed a durable platelet response rate of 23% at week 25, compared to 0% for those on placebo. The drug also enabled faster and longer-lasting platelet responses.
Wayrilz works through multi-immune modulation, targeting various pathways in the immune system, addressing the root causes of ITP rather than just symptoms. Its novel mechanism offers hope for patients with limited options.
Caroline Kruse, President and CEO of the Platelet Disorder Support Association, highlighted the importance of this new therapy for patients and families coping with the physical and emotional challenges of ITP.
Brian Foard, Sanofi’s Executive Vice President and Head of Specialty Care, expressed optimism about Wayrilz becoming a preferred treatment option due to its unique approach and clinical benefits.
The LUNA 3 trial also noted improvements in patients’ quality of life across multiple measures. Common side effects included diarrhea, nausea, headache, abdominal pain, and COVID-19 infection.
Dr. David Kuter from Massachusetts General Hospital underscored the unmet needs in ITP treatment and welcomed Wayrilz as a valuable new option, especially for patients who do not respond to steroids or other therapies.
Wayrilz was previously approved in the United Arab Emirates and is under regulatory review in the European Union and China. It holds Fast Track and Orphan Drug Designations from the FDA, including for other rare diseases like warm autoimmune hemolytic anemia, IgG4-related disease, and sickle cell disease.
Sanofi will offer patient support through its HemAssist program, helping patients with access, insurance navigation, financial assistance, and education throughout their treatment journey.
This approval represents a significant advancement in the management of ITP, providing a novel, targeted therapy for patients with persistent disease.