Sanofi’s Wayrilz approved as the first BTK inhibitor to treat immune thrombocytopenia

Sanofi announces that the European Commission has approved Wayrilz, which is a novel, oral, reversible, Bruton’s tyrosine kinase inhibitor, as a new treatment for immune thrombocytopenia in adult patients who are refractory to other treatments. In addition, this follows the positive opinion by the European Medicines Agency’s Committee for Medicinal Products for Human Use.

“ITP is caused by complex immune system dysregulation leading to low platelet counts, bleeding and other often overlooked symptoms that can affect both physical and mental health, significantly impacting quality of life,” said Waleed Ghanima, MD, Head of Research and a Consultant Hematologist at Østfold Hospital, Norway. “The traditional approach to disease management focuses on restoring platelet counts and reducing bleeding risk, but patients may still experience other symptoms. Wayrilz offers a new approach, targeting the underlying cause of ITP through multi-immune modulation to help address the multi-faceted burden of this disease.”

“The approval of Wayrilz in the EU for the treatment of ITP underscores Sanofi’s commitment to leveraging our knowledge of the immune system to develop innovative treatments that make a meaningful impact on people living with rare and inflammatory diseases,” said Brian Foard, Executive Vice President, Head of Specialty Care at Sanofi. “Wayrilz has a differentiated mechanism of action, enabling multi-immune modulation to address the underlying pathology of ITP, allowing patients to benefit from an advanced treatment to help manage their disease.”

Note that this approval of Wayrilz is based on the pivotal LUNA 3 phase 3 study in which Wayrilz met the primary and secondary endpoints, demonstrating a positive impact on sustained platelet counts as well as other ITP symptoms.

In addition, patients receiving Wayrilz reported an overall 10.6-point improvement in the overall quality of life domain compared to a 2.3-point increase in the placebo arm, based on The Immune Thrombocytopenia Patient Assessment Questionnaire, a clinical tool designed to measure ITP symptoms and impacts. The results of this analysis are descriptive and were not powered for statistical significance.

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Related Topics and Keywords

btk, Sanofi, thrombocytopenia, wayrilz