CONTENTS:
- FDA Approves First-of-its-Kind Treatment for ITP
- ITP and Pregnancy: What You Need to Know Before, During, and After
FDA Approves First-of-its-Kind Treatment for ITP
The U.S. Food and Drug Administration (FDA) has approved a new treatment called Wayrilz (rilzabrutinib) for adults with persistent or chronic immune thrombocytopenia (ITP) who have not responded adequately to other treatments.
Sanofi’s Wayrilz is the first Bruton’s tyrosine kinase (BTK) inhibitor approved for the treatment of ITP. As a novel, oral, and reversible BTK inhibitor, Wayrilz works by modulating multiple parts of the immune system involved in ITP, helping to address the underlying causes of ITP.
The approval was based on a large clinical study called LUNA 3, in which Wayrilz met the primary and secondary endpoints, showing a positive impact on sustained platelet counts and other ITP symptoms. Key outcomes included a faster time to first platelet response, a longer duration of response, a reduction in the need for rescue therapies, and meaningful improvements in patient-reported outcomes like physical fatigue and improved quality of life.
"Traditionally, immune thrombocytopenia management has focused on restoring platelet counts and reducing bleeding risk, which for some patients may result in suboptimal responses, persistent symptoms, or unacceptable treatment complications," said PDSA Medical Advisor David Kuter, MD, Director of Clinical Hematology at Massachusetts General Hospital and Professor of Medicine at Harvard Medical School, study author. "Through multi-immune modulation, Wayrilz can offer a new option for patients, including those who fail steroids or do not respond to existing treatment."
The most common adverse reactions of Wayrilz are diarrhea, nausea, headache, abdominal pain, and COVID-19.
ITP and Pregnancy: What You Need to Know Before, During, and After
Pregnancy with ITP can feel overwhelming—but it doesn't have to be. In this episode of PDSA's podcast, Bruised but Not Broken: Living with ITP, two leading experts—Dr. Terry Gernsheimer and Dr. Edith Cheng—share what women with ITP need to know before, during, and after pregnancy. From coordinating care between hematologists and obstetricians to understanding delivery options and postpartum concerns, this conversation is packed with reassurance, real-world examples, and actionable guidance. Whether you're planning a pregnancy or already expecting, this episode offers clarity, confidence, and hope.