European Commission approves Enjaymo®
(sutimlimab) for treatment of hemolytic anemia in adult patients
with cold agglutinin disease
- Enjaymo is the first-and-only
approved therapeutic option approved for hemolytic anemia in adult
patients with cold agglutinin disease
Paris,
November 17,
2022. The European Commission (EC) has granted marketing
authorization for Enjaymo® (sutimlimab) for the treatment of
hemolytic anemia in adult patients with cold agglutinin disease
(CAD), a rare, serious, and chronic autoimmune hemolytic anemia,
where the body’s immune system mistakenly attacks healthy red blood
cells and causes their rupture, known as hemolysis.
Dietmar Berger, MD,
PhDChief Medical Officer, Global Head of Development at
Sanofi“This approval highlights our ambition to develop first- and
best-in-class medicines that transform people’s lives. Up until
now, patients in Europe had to rely on a combination of cold
avoidance, blood transfusions and off-label treatments to manage
their disease. The approval of Enjaymo by the European Commission
provides patients, for the first time, with access to a therapy
that can make a meaningful difference in the treatment and daily
experience of living with CAD.”
Enjaymo is currently the only approved treatment
for CAD and is a first-in-class humanized monoclonal antibody that
is designed to selectively target and inhibit the classical
complement pathway specific serine protease, C1s. It will be
available as a 50mg/mL solution for infusion.
Alexander
Röth,
MDDepartment of Hematology and Stem Cell Transplantation,
University Hospital, University of Duisburg-Essen, Germany“Coupled
with diagnostic journeys that can last years, the impact of fatigue
on quality of life in CAD is often debilitating and is comparable
to conditions such as cancer-related anemia and other autoimmune
disorders. Clinicians now have a much-needed therapeutic option to
offer to their patients.”
About the CADENZA and CARDINAL Clinical
Trials
The EC approval is based on data from two Phase
3 clinical trials: CADENZA, a double-blind, placebo-controlled
clinical trial of adults with CAD without a recent history of blood
transfusion (within the past 6 months), and CARDINAL, a 26-week
open label, single-arm pivotal study in patients with CAD who have
had a recent blood transfusion.
In the CADENZA Part A trial, eligible patients
were randomized 1:1 to receive a fixed weight-based dose (6.5g or
7.5g) of Enjaymo or placebo via intravenous infusion on Day 0, Day
7, and then once every other week up to Week 26. The open-label
Part B of the study assessed long-term safety as well as durability
of response to Enjaymo in patients with CAD. In the CADENZA Part A
study, Enjaymo met its primary composite endpoint and all secondary
endpoints and demonstrated inhibition of hemolysis, increase in
hemoglobin levels, and clinically meaningful improvement in The
Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue
scores. Enjaymo demonstrated an acceptable safety profile and was
generally well-tolerated. 96% of patients in the Enjaymo group and
100% of patients in the placebo group experienced at least one
treatment emergent adverse event (TEAE). Headache (22.7% vs 10.0%),
hypertension (22.7% vs 0%), rhinitis (18.2% vs 0%), Raynaud
phenomenon (18.2% vs 0%), and acrocyanosis (13.6% vs 0%) were
reported more frequently in Enajymo-treated patients compared with
placebo.
In the CARDINAL Part A trial, patients received
a fixed weight-based dose (6.5g or 7.5g) of Enjaymo via intravenous
infusion on Day 0, Day 7, and then once every other week up to Week
26. Part B of the study evaluated the long-term safety as well as
durability of response to Enjaymo in patients with CAD over a
2-year follow up. In the CARDINAL Part A study, the efficacy of
Enjaymo was assessed based on the achievement of a primary
composite endpoint (Hb≥12 g/dL or an increase of at least 2 g/dL;
no blood transfusion or prohibited medications from Weeks 5 through
26) and different secondary endpoints, including improvements in
hemoglobin, normalization of bilirubin, and FACIT-fatigue score.
The most common adverse reactions occurring in 10% or more of
patients were respiratory tract infection, viral infection,
diarrhea, dyspepsia, cough, arthralgia, arthritis, and peripheral
edema. Serious adverse reactions were reported in 13% (3/24) of
patients who received Enjaymo. These serious adverse reactions were
streptococcal sepsis and staphylococcal wound infection (n=1),
arthralgia (n=1), and respiratory tract infection (n=1).
About Enjaymo® (sutimlimab) Enjaymo is a
humanized monoclonal antibody that is designed to selectively
target and inhibit C1s in the classical complement pathway, which
is part of the innate immune system. By blocking C1s, Enjaymo
inhibits the activation of the complement cascade in the immune
system and inhibits C1-activated hemolysis in CAD to prevent the
abnormal destruction of healthy red blood cells. Enjaymo does not
inhibit the lectin and alternative pathways. Enjaymo was approved
by the US Food and Drug Administration (FDA) in February 2022 as
the first and only treatment indicated to decrease the need for red
blood cell transfusion due to hemolysis in adults with CAD. The
Japanese Ministry of Health, Labor and Welfare approved Enjaymo in
June 2022. The European Medicines Agency (EMA) also made the
decision to maintain orphan designation.
About cold agglutinin disease Cold agglutinin
disease (CAD) is a rare type of autoimmune hemolytic anemia, where
part of the body’s immune system mistakenly destroys healthy red
blood cells (hemolysis). CAD impacts the lives of an estimated
12,000 people in the US, Europe, and Japan and is associated with
profound fatigue and increased risk of thromboembolic events and
mortality.
About SanofiWe are an innovative global
healthcare company, driven by one purpose: we chase the miracles of
science to improve people’s lives. Our team, across some 100
countries, is dedicated to transforming the practice of medicine by
working to turn the impossible into the possible. We provide
potentially life-changing treatment options and life-saving vaccine
protection to millions of people globally, while putting
sustainability and social responsibility at the center of our
ambitions.
Sanofi is listed on EURONEXT: SAN and NASDAQ:
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