A new therapy called rilzabrutinib has shown significant benefits in a recent phase 3 study presented at the American Society of Hematology (ASH) Annual Meeting in December 2024.
What is Rilzabrutinib?
Rilzabrutinib is an oral medication being studied as a treatment for ITP. It works by targeting a protein called BTK (Bruton’s tyrosine kinase), which plays a role in the immune system’s overactivity in ITP. By calming this response, rilzabrutinib helps increase platelet counts, reduce bleeding, and improve quality of life.
Key Findings from the Study
The LUNA 3 study tested rilzabrutinib in adults with persistent or chronic ITP. Here are the main results:
- Platelet Response: 65% of patients taking rilzabrutinib saw an improvement in their platelet levels, compared to 33% on a placebo (a pill with no active ingredients).
- Durable Response: 23% of patients on rilzabrutinib maintained healthy platelet levels for at least 8 out of the last 12 weeks of the study, compared to none in the placebo group.
- Quick Results: Most patients who responded to rilzabrutinib saw their platelet levels rise within just 15 days.
- Less Bleeding: Patients on rilzabrutinib had fewer bleeding episodes, which is a significant relief for those at risk of dangerous bleeds.
- Better Fatigue Levels: Many patients reported feeling less tired and more energetic.
The study also showed a reduced need for emergency treatments like platelet transfusions or steroids, which can cause significant side effects when used long-term.
Dr David Kuter, a leading haematologist and one of the study’s authors, shared his thoughts on the findings:
“People living with immune thrombocytopenia who cannot tolerate or do not respond to medications aimed at raising platelet counts are at risk of uncontrolled bleeding and often endure side effects from steroids and other available therapies. A significant percentage of these patients also suffer from severe fatigue and an impaired quality of life. I’m encouraged by the robust therapeutic effects I’ve seen in patients of the LUNA 3 study across all aspects of the disease, including clinically meaningful and sustained improvements in platelet count, quality of life metrics, reduction in bleeding, and a favourable safety profile.”
What About Side Effects?
Rilzabrutinib was well-tolerated by most patients. Common side effects included mild to moderate diarrhea, nausea, headaches, and abdominal pain. These side effects were generally manageable and occurred at similar rates as in the placebo group.
What Does This Mean for People with ITP?
If approved, rilzabrutinib could be the first of its kind—a BTK inhibitor specifically for ITP. For people who haven’t responded well to current treatments or who struggle with their side effects, this medication offers hope for better platelet control and an improved quality of life.
What’s Next?
Rilzabrutinib is currently under review by regulatory agencies in the US and Europe, with a decision expected by late 2025. While it’s not yet available, these promising results bring optimism for future treatment options.
What is ITP?
ITP is a rare autoimmune condition where the immune system mistakenly attacks and destroys platelets, the blood cells that help with clotting. This can lead to excessive bruising, bleeding, and fatigue, significantly impacting a person’s daily life. Current treatments often include steroids, immunotherapy, or platelet-boosting drugs, but not all patients respond well to these.
Why This Matters
Living with ITP is about more than managing low platelets—it’s about coping with fatigue, anxiety, and the fear of bleeding. A treatment like rilzabrutinib, which addresses both physical and quality-of-life challenges, could be a game-changer for many.
Stay Informed
As rilzabrutinib moves through the approval process, keep an eye out for updates from patient organisations like ITP Australia and New Zealand.
This breakthrough brings renewed hope to the ITP community and highlights the importance of ongoing research in rare diseases.
Review the published Article here – https://doi.org/10.1182/blood-2024-200279
Review the poster abstract here – Rilzabrutinib ITP Poster Abstract
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Article Source: Sanofi.com