New Phase 3 data presented at WORLDSymposium™
reinforce Nexviazyme® (avalglucosidase alfa) as potential new
standard of care for all people living with late-onset Pompe
disease
Paris,
February 24,
2023. Today, at
WORLDSymposiumTM, data from the Phase 3 COMET study long-term
extension showed sustained treatment effect of Nexviazyme®
(avalglucosidase alfa) over nearly three years in late-onset Pompe
disease (LOPD) patients who were treatment-naïve as well as those
who switched from long-time standard of care, alglucosidase alfa,
during the 96-week extension period. Additionally, a separate
analysis of respiratory function showed clinical benefit over two
years for patients who switched to Nexviazyme regardless of prior
response to alglucosidase alfa.
Priya S.
Kishnani, MDC.L. and Su Chen
Professor of Pediatrics; Medical Director, YT and Alice Chen
Pediatrics Genetics and Genomics Center; and Division Chief,
Medical Genetics, Duke University Medical Center “The results
presented at the 2023 WORLDSymposium continue to build on the data
from the clinical trials on the value and long-term impact
Nexviazyme may provide for a wide variety of people living with
late-onset Pompe disease, from newly diagnosed patients to those
who have been previously treated with alglucosidase alfa. Further,
when examining patients treated with Nexviazyme who switched from
using alglucosidase alfa, these data show clinical benefit both for
patients who were stable on alglucosidase alfa and those who
experienced sub-optimal response or decline.”
Bill SiboldGlobal Head of
Specialty Care, Sanofi“These long-term data over nearly three years
add to the robust body of evidence that consistently shows the
clinical benefit and durability of effect of treatment with
Nexviazyme for both treatment-experienced and treatment-naïve
people living with late-onset Pompe disease. We continue to learn
from our 20 years of experience in Pompe disease and build on our
findings in an effort to advance the standard of care.”
Nexviazyme is a monotherapy approved in the US
and other markets for the treatment of LOPD. It is also approved
for infantile-onset Pompe disease (IOPD) in certain markets outside
of the US.
Nexviazyme maintained treatment effect at 145
weeks
The Phase 3 COMET trial enrolled 100 previously
untreated LOPD patients who were randomized to receive either
Nexviazyme (20 mg/kg) or alglucosidase alfa (20 mg/kg) every two
weeks for 49 weeks during the double-blind primary analysis period.
During the open-label extension period, long-term efficacy and
safety outcomes were assessed up to 145 weeks in patients who
continued their treatment with Nexviazyme (n=51), as well as those
who switched to treatment with Nexviazyme from alglucosidase alfa
(n=44) at the conclusion of the primary analysis period (Week
49).
After nearly three years (145 weeks), changes
from baseline (least squares mean [standard error]) showed:
- Patients who received continuous
Nexviazyme treatment for 145 weeks experienced sustained
improvements in respiratory and motor function, showing a 1.40
(1.21) point improvement in forced vital capacity (FVC)
percent-predicted and an average increase of 20.65 (9.60) meters in
walking distance as measured by the six-minute walk test (6MWT),
respectively, compared to baseline.
- Patients who switched from
alglucosidase alfa to Nexviazyme treatment during the extension
period experienced stabilization of treatment effect, showing a
1.18 (1.32) point improvement in FVC percent-predicted and an
average increase of 0.29 (10.42) meters in walking distance (6MWT),
compared to baseline.
The safety profile during treatment with Nexviazyme
was comparable between both study groups (those who started
Nexviazyme in the primary analysis period and those who switched to
Nexviazyme during the extension period). No new safety signals were
observed in patients who switched from alglucosidase alfa to
Nexviazyme during the extension period. Across both Nexviazyme
treatment groups, five patients discontinued treatment during the
extension period due to six adverse events (AEs); four were
treatment-related (ocular hyperemia, erythema, urticaria,
respiratory distress), and two were non-treatment-related (acute
myocardial infarction, pancreatic adenocarcinoma).
Clinical benefits seen over two years for
patients who switched to Nexviazyme
Subgroup analyses were also performed to assess
respiratory function outcomes for the 44 patients who switched to
Nexviazyme in the long-term extension period of the Phase 3 COMET
study. Patients were divided into two subgroups, responders (n=14)
and non-responders (n=30) based on individual responses to initial
treatment with alglucosidase alfa, as measured by the change in FVC
percent-predicted (ΔFVC %predicted/year) pre- (baseline-week 49)
and post-switch (week 49-week 145). Findings showed clinical
benefits over two years following switch to Nexviazyme regardless
of prior response to alglucosidase alfa:
- Patients who responded to
alglucosidase alfa treatment in the primary analysis period had
increased respiratory function (ΔFVC slope, 4.67±1.28%/yr,
p<0.001), which was maintained throughout the extension period
with Nexviazyme treatment (ΔFVC slope, 0.14±0.94%/yr, p=0.88).
- Patients who did not respond to
alglucosidase alfa treatment in the primary analysis period had
reduced respiratory function while taking alglucosidase alfa (ΔFVC
slope, -2.12±0.87%/yr, p=0.016); however, switching to Nexviazyme
halted this decline in the extension period (ΔFVC slope,
0.15±0.61%/yr, p=0.81).
About Pompe disease
People living with Pompe disease have low levels
of the enzyme acid alpha-glucosidase (GAA), which results in
build-up of glycogen in muscle cells throughout the body, leading
to irreversible damage to skeletal and cardiac muscles.
Pompe disease can present as infantile-onset
Pompe disease (IOPD), the most severe form of the disease with
rapid onset in infancy, or late-onset Pompe disease (LOPD), which
progressively damages muscles over time. If left untreated, IOPD
can lead to heart failure and death within the first year of life,
while people living with LOPD may require mechanical ventilation to
help with breathing or a wheelchair to assist with mobility as the
disease progresses.
About
Nexviazyme (avalglucosidase alfa)
Nexviazyme (avalglucosidase alfa) is an enzyme
replacement therapy (ERT) designed to target the
mannose-6-phosphate (M6P) receptor, the key pathway for uptake and
transport of ERT. Nexviazyme aims to help improve uptake and
enhance glycogen clearance in target tissues with an average
15-fold higher level of M6P moieties as compared to alglucosidase
alfa, the comparator therapy in the pivotal study. Nexviazyme is
approved in multiple markets around the world for the treatment of
people living with Pompe disease, including the United States, the
European Union, Japan, Canada, Switzerland, Australia, Brazil,
Argentina, Taiwan, the United Arab Emirates, South Korea and the
United Kingdom, with specific indications varying by country. In
Europe, the medicine is marketed under the brand name
Nexviadyme.
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: SNY
Media RelationsSandrine
Guendoul | + 33 6 25 09 14 25 |
sandrine.guendoul@sanofi.comKate
Conway | + 1 508 364 4931
| kate.conway@sanofi.com
Investor RelationsEva
Schaefer-Jansen | + 33 7 86 80 56 39
| eva.schaefer-jansen@sanofi.comArnaud
Delépine | + 33 6 73 69
36 93 | arnaud.delepine@sanofi.comCorentine
Driancourt | + 33 6 40 56 92 21 |
corentine.driancourt@sanofi.comFelix
Lauscher | + 1 908 612 7239 |
felix.lauscher@sanofi.comTarik Elgoutni| + 1 617
710 3587 | tarik.elgoutni@sanofi.comNathalie
Pham | + 33 7 85 93 30 17 |
nathalie.pham@sanofi.com
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