Sanofi wins approval for first Bruton’s tyrosine kinase inhibitor in immune thrombocytopenia

1. The FDA approved rilzabrutinib, branded Wayrilz, as the first BTK (Bruton’s tyrosine kinase) inhibitor for adults with ITP (immune thrombocytopenia).

2. The oral therapy showed durable platelet responses in Phase 3 and enters the market with a 17,500 dollar monthly list price.

On August 29, the FDA approved Wayrilz, also known as rilzabrutinib, for adult patients with ITP, according to Reuters. Sanofi highlighted the approval as a first-in-class BTK inhibitor for this rare autoimmune bleeding disorder, noting in its press release that Phase 3 trials demonstrated meaningful platelet count improvements. The FDA added Wayrilz to its notable approvals list on September 2, confirming its place among recent important therapies. The starting price is 17,500 dollars per month, which immediately sets the stage for payer negotiations and access challenges. For hematologists, the convenience of an oral therapy is appealing, especially after steroids, TPO receptor agonists, or splenectomy have failed. Safety considerations will remain important, with BTK inhibition linked to bleeding and infection risks in other settings. Clinics will likely monitor patients closely during the first months to understand how tolerability compares to existing options. Some experts believe the drug could reshape second-line therapy if real-world durability mirrors trial results. Others caution that cost will be a limiting factor, especially without robust assistance programs. Earlier this week, several teaching hospitals began updating treatment pathways to reflect the new approval. Patients, meanwhile, may see this as a welcome alternative to long-term steroids or invasive procedures. For clinicians, Wayrilz represents not just another tool but the opening of a new drug class in a difficult disease. And it may influence how quickly other BTK inhibitors move forward in hematology.

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