Press Release

Sanofi’s Wayrilz recommended for EU approval by the
CHMP to treat immune thrombocytopenia
• Recommendation based on LUNA 3 phase 3 study demonstrating rapid and durable
platelet response and significant improvements in bleeding, quality of life measures, and
other ITP symptoms
• If approved, Wayrilz will be the first BTK inhibitor for ITP in the EU, targeting the root
cause of the disease through multi-immune modulation

Paris, October 17, 2025. The European Medicines Agency’s Committee for Medicinal Products
for Human Use (CHMP) has adopted a positive opinion recommending the approval of Wayrilz
(rilzabrutinib) as a new treatment for immune thrombocytopenia (ITP) in adult patients who are
refractory to other treatments. A final decision is expected in the coming months.

“The CHMP’s recommendation of Wayrilz for approval is a positive step forward for
people in the EU living with ITP who continue to experience symptoms that impact
their daily living despite treatment with certain other therapies,” said Brian Foard,
Executive Vice President, Head of Specialty Care at Sanofi. “Through multi-immune
modulation, Wayrilz shows promise in addressing the root cause of ITP – complex
immune system dysregulation, reinforcing Sanofi’s commitment to finding novel ways
to address unmet patient needs in rare and immunological diseases.”

The positive CHMP opinion is based on the pivotal LUNA 3 phase 3 study (clinical study identifier:
NCT04562766), presented at the 66th American Society of Hematology Annual Meeting and
Exposition in December 2024 and also published in Blood. Wayrilz met both the primary and
secondary endpoints, making LUNA 3 the first phase 3 study to show a positive impact on
sustained platelet counts, bleeding, and other ITP symptoms with an oral, reversible, Bruton’s
tyrosine kinase (BTK) inhibitor.

Wayrilz has already been approved in the US and the United Arab Emirates. In addition to the
EU, Wayrilz is currently under regulatory review for ITP in China. It received fast track and
orphan drug designations (ODD) in the US for ITP, with similar orphan designations in the EU
and Japan. In other indications under investigation, the US Food and Drug Administration (FDA)
granted Wayrilz ODD for three additional rare diseases, including warm autoimmune hemolytic
anemia (wAIHA), IgG4-related disease (IgG4-RD), and sickle cell disease (SCD). Wayrilz also
received FDA fast track designation and EU orphan designation in IgG4-RD.

About the LUNA 3 study
LUNA 3 (clinical study identifier: NCT04562766) was a randomized, multicenter, phase 3 study
evaluating the efficacy and safety of Wayrilz vs. placebo in adult and adolescent patients with
persistent or chronic ITP. Patients received either oral Wayrilz 400 mg twice a day or placebo
through a 12- to 24-week double-blind treatment period, followed by a 28-week open-label
treatment period, and then a four-week safety follow-up or long-term extension period. The
adolescent part of the study is ongoing. The primary endpoint for the EU is the proportion of
adult participants able to achieve platelet counts at or above 50,000/µL for at least eight out of
the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy.
Secondary endpoints included time to platelet response (platelet count ≥50 x
109/L or between 30 x 109/L and <50 x 109/L and at least doubled from baseline in absence of
rescue therapy), number of weeks maintaining a specific platelet response (i.e., doubled or
within range), rescue therapy use, physical fatigue score, and bleeding score as assessed by
change from baseline in Idiopathic Thrombocytopenic Purpura Bleeding Scale (IBLS) assessment
at Week 25.



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About Wayrilz
Wayrilz (rilzabrutinib) is the first BTK inhibitor for ITP that helps address the root cause of disease
through multi-immune modulation. This innovative therapy is approved in the US for adults with
persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to
a previous treatment. Additionally, Wayrilz is approved in the United Arab Emirates for adult
patients with persistent or chronic ITP who have had an insufficient response or intolerance to a
previous treatment. BTK, expressed in B cells, macrophages, and other innate immune cells,
plays a critical role in multiple immune-mediated disease processes and inflammatory pathways.
With the application of Sanofi’s TAILORED COVALENCY® technology, Wayrilz can selectively
inhibit the BTK target.

Wayrilz is being studied across a variety of rare diseases, including wAIHA, IgG4-RD, and SCD.
These additional indications are currently under investigation and have not been approved by
regulatory authorities.

About ITP
ITP is a disease of complex immune dysregulation that causes low platelet counts
(<100,000/µL), resulting in a variety of bleeding symptoms and thromboembolism risk. Beyond
bruising and bleeding, which can include potentially life-threatening episodes like intracranial
hemorrhage, people living with ITP may experience reduced quality of life, including physical
fatigue and cognitive impairment.

About Sanofi
Sanofi is an R&D driven, AI-powered biopharma company committed to improving people’s lives
and delivering compelling growth. We apply our deep understanding of the immune system to
invent medicines and vaccines that treat and protect millions of people around the world, with
an innovative pipeline that could benefit millions more. Our team is guided by one purpose: we
chase the miracles of science to improve people’s lives; this inspires us to drive progress and
deliver positive impact for our people and the communities we serve, by addressing the most
urgent healthcare, environmental, and societal challenges of our time.
Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY

Media Relations
Sandrine Guendoul | + 33 6 25 09 14 25 | sandrine.guendoul@sanofi.com
Evan Berland | +1 215 432 0234 | evan.berland@sanofi.com
Léo Le Bourhis | + 33 6 75 06 43 81 | leo.lebourhis@sanofi.com
Victor Rouault | + 33 6 70 93 71 40 | victor.rouault@sanofi.com
Timothy Gilbert | + 1 516 521 2929 | timothy.gilbert@sanofi.com
Léa Ubaldi | +33 6 30 19 66 46 | lea.ubaldi@sanofi.com

Investor Relations
Thomas Kudsk Larsen | +44 7545 513 693 | thomas.larsen@sanofi.com
Alizé Kaisserian | + 33 6 47 04 12 11 | alize.kaisserian@sanofi.com
Felix Lauscher | +1 908 612 7239 | felix.lauscher@sanofi.com
Keita Browne | +1 781 249 1766 | keita.browne@sanofi.com
Nathalie Pham | +33 7 85 93 30 17 | nathalie.pham@sanofi.com
Tarik Elgoutni | +1 617 710 3587 | tarik.elgoutni@sanofi.com
Thibaud Châtelet | +33 6 80 80 89 90 | thibaud.chatelet@sanofi.com
Yun Li | +33 6 84 00 90 72 | yun.li3@sanofi.com


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