Sanofi’s oral reversible Bruton’s tyrosine kinase (BTK) inhibitor, Wayrilz (rilzabrutinib), has secured approval from the US Food and Drug Administration (FDA) for adult patients with persistent or chronic immune thrombocytopenia (ITP) who have not responded sufficiently to previous treatments.

Wayrilz achieved both primary and secondary goals in the LUNA 3 Phase III trial, which backed the approval process by the agency.

The therapy demonstrated a positive influence on sustained platelet counts and alleviation of other symptoms associated with ITP.

The trial assessed the safety and efficacy of the therapy compared to a placebo in 202 adults.

Those who showed a response in platelet count by the 12th week were allowed to continue in the study for a complete double-blind period of 24 weeks, involving 64% of subjects in the therapy arm and 32% in the placebo arm.

Subjects on Wayrilz experienced a durable platelet response at week 25, a faster time to first platelet response and a longer duration of platelet response compared to those on placebo.

They also reported an improvement in health-related quality of life measures, as determined by the Immune Thrombocytopenia Patient Assessment Questionnaire. However, these findings were descriptive and not statistically powered for significance.

Sanofi Specialty Care head and executive vice-president Brian Foard stated: “With its differentiated mechanism of action, Wayrilz has the potential to become a treatment of choice for immune thrombocytopenia patients who have not responded to a prior therapy.

“Its multi-immune modulation approach shows promise in addressing the key drivers of immune thrombocytopenia, which aligns with Sanofi's commitment to adapting and evolving therapeutic solutions to help tackle ongoing unmet patient needs.”

Sanofi's Wayrilz had previously been approved in the United Arab Emirates for the same indication and is being reviewed by regulatory authorities for ITP in China and the European Union.

The European Medicines Agency has granted orphan designation for Wayrilz in IgG4-related disease (IgG4-RD).

The development of Wayrilz has been enhanced by Sanofi’s TAILORED COVALENCY technology, which allows for the selective inhibition of the BTK target.