- Approved for the treatment of the full spectrum of both late-onset Pompe disease and infantile-onset Pompe disease
- First new treatment option approved for the Pompe community in
of alglucosidase alfa, branded Myozyme®.
Senior Consultant and Professor, Friedrich-Baur-Institute,
"The approval of Nexviadyme in
Addressing an unmet need for people living with Pompe disease
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 with LOPD may require mechanical ventilation to help with breathing or a wheelchair to assist with mobility as the disease progresses.
Nexviadyme now approved in many countries around the world
Nexviadyme is approved in multiple markets around the world for the treatment of certain people living with Pompe disease, including the
In
Executive Vice President, Specialty Care,
"For more than two decades, we've been working with the community and leveraging our scientific expertise to improve care for people living with Pompe disease. We strongly believe in the meaningful clinical benefits of this medicine as a new standard of care and will work hard to ensure the broadest possible access in
Nexviadyme, a new ERT for late-onset Pompe disease and infantile-onset Pompe Disease
Positive outcomes in key disease burden measures
In a robust clinical development program, Nexviadyme demonstrated clinically meaningful differences in key areas of disease burden for people living with late-onset Pompe disease and infantile-onset Pompe disease.
Results from the COMET study comparing Nexviadyme to alglucosidase alfa in LOPD at 49 weeks included:
- Patients treated with Nexviadyme showed a 2.9% improvement from baseline (SE=0.9) in forced vital capacity (FVC) percent-predicted, a key measure of respiratory function and the study's primary endpoint, which was 2.4% points greater as compared to the change with alglucosidase alfa. This difference exceeded the non-inferiority margin (p=0.0074; 95% CI, -0.13, 4.99). Statistical superiority was narrowly missed (p=0.06).
- Patients treated with Nexviadyme walked 32.2 meters farther (SE=9.9) compared to baseline in the 6-minute walk test (6MWT), a key secondary endpoint, which was 30 meters farther (p=0.040; 95% CI, 1.33, 58.69) than the change with alglucosidase alfa. Formal statistical testing for all secondary endpoints was not conducted.
Results from the Mini-COMET study evaluating Nexviadyme in IOPD patients showed improvement or stabilization at six months in efficacy outcomes, the trial's secondary objective, of gross motor function measure (GMFM-88), quick motor function test (QMFT), pediatric evaluation of disability index (Pompe-PEDI), left ventricular mass z-score (LVMZ), and eyelid position measurements in patients previously declining or insufficiently controlled with alglucosidase alfa.
A pooled safety analysis from four clinical studies found serious adverse reactions reported in patients treated with Nexviadyme included chills (1.4%), headache, dyspnoea, respiratory distress, nausea, skin discoloration, chest discomfort, pyrexia, blood pressure increased, body temperature increased, heart rate increased, and oxygen saturation decreased (0.7% each). Additionally, hypersensitivity reactions (43.5%), anaphylaxis (1.4%) and infusion-associated reactions (26.1%) were reported. The most frequently reported adverse drug reactions (ADRs) (>5%) were pruritus (9.4%), rash (8%), headache (7.2%), urticaria (6.5%), fatigue (6.5%), nausea (5.8%), and chills (5.1%).
Mechanism of action designed for increased uptake
People living with Pompe disease have low levels of the enzyme acid alpha-glucosidase (GAA), which results in build-up of glycogen, leading to irreversible damage to skeletal and cardiac muscles. Nexviadyme is specifically designed to target the mannose-6-phosphate (M6P) receptor, the key pathway for cellular uptake of ERT and transport to the lysosome, and has an average 15-fold higher level of M6P moieties as compared to alglucosidase alfa. Nexviadyme aims to help improve uptake and enhance glycogen clearance in target tissues as compared to alglucosidase alfa, which was used as the comparator arm in the pivotal Phase 3 COMET study.
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