The European Commission has approved Wayrilz (rilzabrutinib), a novel oral Bruton’s tyrosine kinase (BTK) inhibitor, for adult patients with immune thrombocytopenia (ITP) refractory to other treatments. The move follows a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP). 

Wayrilz represents a new approach to ITP by targeting the disease at its root through multi-immune modulation, acting across different immune pathways. 

“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 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.” 

Sanofi, the drug’s manufacturer, hailed the EU approval as a milestone for patients with rare and inflammatory diseases. 

“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.” 

The most common side effects were diarrhea, nausea, headache, abdominal pain, and COVID-19. 

Wayrilz has already received approval in the US and UAE and is under review in Japan and China. It has orphan drug designation (ODD) and fast track status in multiple countries, with ongoing investigations in additional rare diseases including warm autoimmune hemolytic anemia, IgG4-related disease, and sickle cell disease. 

The LUNA 3 study involved adults and adolescents with persistent or chronic ITP, receiving Wayrilz 400 mg twice daily or placebo for 12–24 weeks, followed by a 28-week open-label period and long-term safety follow-up.  

The primary endpoint was achieving platelet counts ≥50,000/μL for at least eight of the last 12 weeks without rescue therapy. Secondary endpoints included time to platelet response, duration of response, need for rescue therapy, and changes in physical fatigue and bleeding scores.