FDA approves
Nexviazyme®
(avalglucosidase
alfa-ngpt),
an important new treatment option
for late-onset
Pompe disease
- Approval is based on positive Phase 3 data demonstrating
improvements in key disease burden measures and establishing its
safety profile
- Nexviazyme specifically targets the M6P receptor, the key
pathway for enzyme replacement therapy, to effectively clear
glycogen build-up in muscle cells
PARIS –
August 6, 2021 -
The U.S. Food and Drug Administration (FDA) has approved
Nexviazyme® (avalglucosidase alfa-ngpt) for the treatment of
patients one year of age and older with late-onset Pompe disease, a
progressive and debilitating muscle disorder that impairs a
person’s ability to move and breathe. Nexviazyme is an enzyme
replacement therapy (ERT) designed to specifically target the
mannose-6-phosphate (M6P) receptor, the key pathway for cellular
uptake of enzyme replacement therapy in Pompe disease. Nexviazyme
has been shown in clinical trials to provide patients with
improvements in respiratory function and walking distance. “Pompe
disease is a debilitating and progressive condition that
significantly inhibits mobility and breathing,” said Bill Sibold,
Executive Vice President of Sanofi Genzyme. “For decades, we’ve
made it our responsibility to research how to target the M6P
receptor, the key pathway for cellular uptake of enzyme replacement
therapy. Nexviazyme is a potential new standard of care for people
living with late-onset Pompe disease and delivers on our promise to
pursue medicines for patients living with rare diseases.”Pompe
disease affects an estimated 3,500 people in the United States and
can present as infantile-onset Pompe disease (IOPD), the most
severe form of Pompe disease with rapid onset in infancy, and
late-onset Pompe disease (LOPD), which progressively damages
muscles over time. LOPD symptoms may present at any age. However,
due to the wide spectrum of clinical presentations and progressive
nature of the disease, it can take seven to nine years before
patients receive an accurate diagnosis. As the disease progresses,
people with LOPD may require mechanical ventilation to help with
breathing or a wheelchair to assist with mobility.
Targeted delivery to clear glycogen in
muscle cells
Pompe disease is caused by a genetic deficiency
or dysfunction of the lysosomal enzyme acid alpha-glucosidase
(GAA), which results in build-up of complex sugars (glycogen) in
muscle cells throughout the body. The accumulation of glycogen
leads to irreversible damage to the muscles, including the
diaphragm that supports respiratory function and skeletal muscles
that affect mobility, functional endurance and breathing.
The key pathway to
transport GAA enzyme into the lysosomes in the cell is through the
M6P receptor. Nexviazyme is specifically designed to target M6P to
improve cellular enzyme uptake and enhance glycogen clearance in
target tissues with an approximate 15-fold increase in M6P content
compared to alglucosidase alfa, the comparator arm in the pivotal
study.
Nexviazyme demonstrated
improvements in pivotal study
Nexviazyme has demonstrated improvements for
people living with late-onset Pompe disease. In the pivotal Phase 3
trial (COMET), Nexviazyme showed improvements in respiratory
function and walking distance measures in people with LOPD and
established its safety profile.
“Nexviazyme is a new and exciting therapeutic
option for people with late-onset Pompe disease,” said Mazen M.
Dimachkie, MD, FAAN, FANA, Professor of Neurology, Chief of the
Neuromuscular Division and Executive Vice Chair of the Department
of Neurology at the University of Kansas Medical Center. “The Phase
3 study results showed meaningful improvements in respiratory
function and walking distance, which are impactful in this serious
condition.”
Results from the COMET study comparing
Nexviazyme to alglucosidase alfa in LOPD included:
- When compared to baseline, patients
treated with Nexviazyme had a 2.9-point improvement (SE=0.9) in
forced vital capacity (FVC) percent-predicted at Week 49, the
study’s primary endpoint. Patients treated with Nexviazyme had a
2.4-point greater improvement in FVC percent-predicted compared to
patients treated with alglucosidase alfa at Week 49 meeting the
measurement of non-inferiority (p=0.0074; 95% CI, -0.13, 4.99).
Statistical superiority of Nexviazyme over alglucosidase alfa was
not achieved (p=0.06).
- A key secondary endpoint in the
trial measured functional endurance with the 6-minute walk test
(6MWT). When compared to baseline, patients treated with Nexviazyme
walked 32.2 meters farther (SE=9.9) at Week 49. Patients treated
with Nexviazyme walked 30 meters farther (95% CI, 1.33, 58.69) than
patients treated with alglucosidase alfa at Week 49. Per the
hierarchy of the study protocol, formal statistical testing for all
secondary endpoints was not conducted.
- During the double-blind
active-controlled period of 49 weeks, serious adverse reactions
were reported in two (2%) patients treated with Nexviazyme and in
three (6%) patients treated with alglucosidase alfa. The most
frequently reported adverse reactions (>5%) in
Nexviazyme-treated patients were headache, pruritus (itching
sensation), nausea, hives and fatigue.
- Infusion associated reactions were
reported in 13 (25%) of the Nexviazyme-treated patients and in 16
(33%) of patients treated with alglucosidase alfa. Infusion
associated reactions reported in more than one patient on
Nexviazyme were mild to moderate and included headache, diarrhea,
itching, hives, and rash. None of the infusion associated reactions
were severe.
Nexviazyme, a new ERT for
late-onset Pompe
disease
Nexviazyme is administered as a monotherapy ERT
every two weeks. The recommended dose is based on body weight (20
mg/kg for LOPD patients ≥30 kg or 40 mg/kg for LOPD patients <30
kg) and is administered incrementally via intravenous infusion.
Nexviazyme is expected to be available in the U.S. in the coming
weeks.
As part of our commitment to ensure treatment
access and affordability for innovative therapies, Sanofi has
decided to price Nexviazyme the same as alglucosidase alfa, the
only other FDA-approved therapy for the treatment of Pompe disease
and the comparator arm in the pivotal study. Sanofi’s
CareConnectPSS Patient Support Services (1-800-745-4447, Opt. 3)
provides personalized support for people and their families
impacted by Pompe disease, including patients transitioning to
Nexviazyme.
The FDA approval follows a priority review by
the FDA, which is reserved for medicines that, if approved, would
represent significant improvements in safety or efficacy in
treating serious conditions. Previously, Nexviazyme received FDA
Breakthrough Therapy and Fast Track designations for the treatment
of people with Pompe Disease. The European Medicines Agency's (EMA)
Committee for Medicinal Products for Human Use (CHMP) has adopted a
positive opinion for avalglucosidase alfa. While Sanofi is pleased
with the CHMP’s recognition of the clinically meaningful
improvements demonstrated in the avalglucosidase alfa development
program, the CHMP has also rendered an opinion that avalglucosidase
alfa does not qualify as a New Active Substance (NAS). As a result,
Sanofi has requested a re-examination of the CHMP opinion in
relation to the NAS conclusion. Sanofi also filed avalglucosidase
alfa in Japan in January 2021. The safety and efficacy of
avalglucosidase alfa has not been fully evaluated by any regulatory
authority outside of the U.S.
About Sanofi
Sanofi is dedicated to supporting people through
their health challenges. We are a global biopharmaceutical company
focused on human health. We prevent illness with vaccines, provide
innovative treatments to fight pain and ease suffering. We stand by
the few who suffer from rare diseases and the millions with
long-term chronic conditions.
With more than 100,000 people in 100 countries,
Sanofi is transforming scientific innovation into healthcare
solutions around the globe.
Media Relations
ContactsAshleigh KossTel: +1 (908)
205-2572Ashleigh.Koss@sanofi.com
Sally BainTel: +1 (781)
264-1091Sally.Bain@sanofi.com
Investor Relations Contacts
ParisEva Schaefer-JansenArnaud DelepineNathalie Pham
Investor Relations Contacts North
AmericaFelix LauscherFara BerkowitzSuzanne Greco
Tel.: +33 (0)1 53 77 45
45investor.relations@sanofi.comhttps://www.sanofi.com/en/investors/contact
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