Sanofi announces new data highlight innovation from pipeline in rare diseases

Sanofi releases their new data from 18 abstracts, including five oral presentations which will be presented at the 33rd congress of the International Society on Thrombosis and Haemostasis.

Christopher Corsico, MD
Global Head of Development
“Our commitment to rare blood diseases drives us to find innovative solutions for the persistent unmet needs of patients and caregivers. Our data highlight the benefits of our hemophilia treatments and the potential of our reversible oral BTK inhibitor, rilzabrutinib, which could be transformative in rare diseases marked by complex immune dysregulation and inflammation.”

Note that the new findings from the LUNA 3 phase 3 study of rilzabrutinib in ITP will be included in an oral presentation highlighting platelet response as defined by International Working Group criteria – a clinically meaningful endpoint used to guide treatment decisions. Two additional poster presentations will detail the first efficacy and safety report from the LUNA 3 open-label period, along with new insights into platelet count variability in patients treated with rilzabrutinib.

The safety and efficacy of rilzabrutinib have not been determined by any regulatory authority. Rilzabrutinib is currently under regulatory review in the US, the EU, and China in ITP. Rilzabrutinib has received orphan drug designation in various parts of the world in several potential indications, including ITP, wAIHA, sickle cell disease, and IgG4-related disease.

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