Expanded approval includes children aged one year and above with stage 2 T1D to delay the onset of stage 3
The US Food and Drug Administration (FDA) has approved the
supplemental biologic license application for Tzield (teplizumab-mzwv), expanding the indication
from eight years and older to as young as one year of age to delay the onset of stage 3 type 1
diabetes (T1D) in patients diagnosed with stage 2 T1D. The approval was granted under a priority
review process and is supported by one-year data from the PETITE-T1D phase 4 study (clinical
study identifier: NCT05757713), evaluating safety and pharmacokinetics in young children.
"This approval opens an important new chapter in diabetes care for young children
with stage 2 type 1 diabetes and their families," said Kimber Simmons, MD, MS,
Associate Professor of Pediatrics at the Barbara Davis Center, Aurora, Colorado, US.
"This is especially important because these children are often at the highest risk of
progressing quickly and without warning. Delaying the onset of stage 3 type 1 diabetes
during the years when management is often most difficult because of a child´s small
size and dependence on caregivers could have a truly meaningful impact for families."
"The autoimmune attack driving this disease often begins early in life, and the burden
that autoimmune T1D poses in this very young population and their families is
significant," said Christopher Corsico, Global Head of Development at Sanofi. "This
approval underscores the importance of targeting the immune system early in
autoimmune type 1 diabetes, aiming to impact its natural progression by delaying the
loss of insulin production in the pancreas."
Tzield is also being reviewed by the FDA for a potential indication to delay the progression of
stage 3 T1D in patients eight years of age and older recently diagnosed with stage 3 T1D.
Tzield is approved in the EU (under the name Teizeild), in the UK, China, Canada, Israel, Saudi
Arabia, the UAE, Kuwait, and Brazil to delay the onset of stage 3 T1D in adults and pediatric
patients eight years and older diagnosed with stage 2 T1D. Other regulatory reviews are
ongoing. Tzield was previously granted FDA breakthrough therapy designation and orphan drug
designation, for medicines that treat rare diseases affecting fewer than 200,000 people in the
US.
PETITE-T1D (clinical study identifier: NCT05757713) is a phase 4 single-arm, non-randomized,
open-label, multi-center study designed to assess the safety and pharmacokinetics of Tzield in
children under eight years diagnosed with stage 2 T1D. Stage 2 T1D is defined by the presence
of two or more T1D-related autoantibodies and abnormal blood sugar levels (dysglycemia).
The study has enrolled 23 participants. The regimen consists of an intravenous infusion of Tzield
once daily for 14 consecutive days. The study duration for each individual may last up to 26
months including screening for eligibility, treatment administration and follow-up.
T1D is a progressive autoimmune disease where the body´s ability to regulate blood sugar
levels is impacted due to the gradual destruction of insulin-producing beta cells by one´s own
immune system. There are four stages to the progression of T1D:
2/3
· In stage 1, the autoimmune attack to the beta cells has started, and this can be detected
by the presence of two or more T1D-related autoantibodies in the blood. During stage 1,
blood sugar levels are in a normal range (normoglycemia). At this stage, T1D is
presymptomatic.
· In stage 2 (also presymptomatic), in addition to the presence of two or more T1D-related
autoantibodies, blood sugar levels become abnormal (dysglycemia) due to the
progressive loss of beta cells / beta-cell function.
· Stage 3 (also known as clinical stage) comes once a significant portion of the beta cells
have been destroyed. At this point, rising blood sugar levels reach the point of clinical
hyperglycemia (which defines diabetes), and many people will start to experience the
classic symptoms that come with the onset of stage 3 T1D: increased thirst, frequent
urination, unexplained weight loss, blurred vision, and generalized fatigue. Management
of stage 3 T1D requires daily and burdensome insulin replacement therapy.
· Stage 4 is defined as long-standing autoimmune T1D, often accompanied by evidence of
chronic diabetic complications, where little to no beta-cell function remains (it has been
estimated that beta-cell mass is reduced by up to 95%). At this point, the T1D-related
autoantibodies might not be present anymore in the blood, as most beta cells have been
rendered useless by the autoimmune attack.
Tzield (teplizumab-mzwv) is a CD3-directed monoclonal antibody. Tzield is the first disease
modifying medicine in autoimmune T1D. It was first approved in the US in November 2022 to
delay the onset of stage 3 T1D in adults and children eight years and older diagnosed with stage
2 T1D. Tzield is also approved in the EU (under the name Teizeild), the UK, China, Canada,
Israel, Saudi Arabia, the UAE, Kuwait, and Brazil to delay the onset of stage 3 T1D in adults and
children eight years and older diagnosed with stage 2 T1D.
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
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