Sanofi Wins FDA Approval for New Blood Disorder Drug Wayrilz
First-in-class BTK inhibitor for immune thrombocytopenia (ITP) projected to achieve multi-billion dollar peak sales.
French pharmaceutical giant Sanofi has secured U.S. Food and Drug Administration (FDA) approval for Wayrilz (rilzabrutinib), its novel treatment for the rare blood disorder chronic immune thrombocytopenia (ITP). The approval marks a significant milestone, making Wayrilz the first Bruton's tyrosine kinase (BTK) inhibitor available for ITP in the United States and bolstering Sanofi's pipeline with a drug forecasted to reach peak sales between €2 billion and €5 billion.
The drug, acquired through Sanofi's $3.7 billion purchase of Principia Biopharma in 2020, represents a new approach to treating ITP, a complex autoimmune disease characterized by low platelet counts that can lead to dangerous bleeding. Unlike existing therapies that primarily focus on raising platelet levels, Wayrilz is designed to address the underlying causes of the disease through a novel mechanism of 'multi-immune modulation.'
“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,” said Brian Foard, Sanofi's Executive Vice President and Head of Specialty Care, in a statement.
The FDA's decision was based on the positive results of the pivotal LUNA 3 phase 3 study. The trial demonstrated that 23% of patients treated with Wayrilz achieved a durable platelet response, compared to 0% in the placebo group. Patients receiving the drug also experienced a faster and longer-lasting response, alongside improvements in quality-of-life measures like physical fatigue.
Dr. David Kuter, Director of Clinical Hematology at Massachusetts General Hospital and a study author, noted the limitations of traditional ITP management. “Wayrilz can offer a new option for patients, including those who fail steroids or do not respond to existing treatment,” he said.
Sanofi is investigating rilzabrutinib for a range of other immune-mediated conditions, including asthma and chronic hives, positioning it as a potential multi-indication blockbuster. The drug is already approved for ITP in the United Arab Emirates and is currently under regulatory review in the European Union and China.