Availability of Sanofi’s Nexviadyme®▼(avalglucosidase alfa) provides NHS with new treatment for Pompe disease
pharmafile | February 8, 2023 | News story | Business Services |
READING, ENGLAND – Wednesday 8th February 2023. Sanofi announced today that healthcare specialists and patients living with Pompe disease will now have access to a new treatment – Nexviadyme®▼ (avalglucosidase alfa)1 – providing the NHS with a NICE approved, long-term enzyme replacement therapy.2
Prior to the availability of avalglucosidase alfa, only one treatment for Pompe disease has been available (alglucosidase alfa, branded Myozyme®).2,3 The current standard of enzyme replacement therapy has been shown to extend overall survival and slow disease progression in Pompe patients compared to the untreated historical cohort.3 However, it is recognised there is a need for improved treatment options that can offer a greater benefit and longer duration of response.2
Dr Duncan Cole
Deputy Director, Clinical Reader in Medical Education, Honorary Consultant in Medical Biochemistry and Metabolic Medicine, University Hospital of Wales and Cardiff University
“Pompe disease is severely debilitating, effecting both quality and length of life. People with Pompe disease need tailored supportive care from multidisciplinary teams of health professionals. There is a well-recognised need for new treatment options for people whose disease is not well controlled on alglucosidase alfa, or if the treatment effect has waned. Avalglucosidase alfa has an improved uptake compared to our only treatment option to date which should get more enzyme into muscle cells and help address a significant unmet need.”
Pompe disease is a rare, inherited, genetic disease affecting approximately 1 in every 40,000 people in the UK.4 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.2 If left untreated, IOPD can lead to heart complications and death, with an average life expectancy of 14 months, while people with LOPD may require invasive or non-invasive ventilation to help with breathing or a wheelchair to assist with mobility as the disease progresses.2 NICE has recommended avalglucosidase alfa for use in the NHS in the full spectrum of both LOPD and IOPD [TA821].2
Positive outcomes in key disease burden measures
In a robust clinical development programme, avalglucosidase alfa demonstrated clinically meaningful differences in key areas of disease burden for people living with LOPD and IOPD.5,6
Results from the Phase III COMET trial comparing avalglucosidase alfa to alglucosidase alfa in LOPD at 49 weeks included:5
- Patients treated with avalglucosidase alfa showed a 2.89% improvement from baseline (SE=0.9), compared to 0.46% with alglucosidase alfa, 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.5 This difference exceeded the non-inferiority margin (p=0.0074; 95% CI, -0.13, 4.99). Statistical superiority was narrowly missed (p=0.06).5
- Patients treated with avalglucosidase alfa walked 32.2 metres farther (SE=9.9) compared to baseline in the 6-minute walk test (6MWT), a key secondary endpoint, which was 30 metres further (p=0.040; 95% CI, 1.33, 58.69) than the change with alglucosidase alfa.5 Formal statistical testing for all secondary endpoints was not conducted.5
Results from the Mini-COMET study evaluating avalglucosidase alfa in IOPD patients showed improvement or stabilisation at six months in efficacy outcomes, the trial’s secondary objective, of gross motor function measure (GMFM-88), quick motor function test (QMFT), paediatric evaluation of disability index (Pompe-PEDI), left ventricular mass index (LVMI), and eyelid position measurements in patients previously declining or insufficiently controlled with alglucosidase alfa.6
The safety profile of avalglucosidase alfa is consistent with the standard of care.1
Rippon Ubhi
General Manager UK & Ireland, Sanofi Specialty Care
“The availability of avalglucosidase alfa in the UK marks another critical milestone in Sanofi’s 20-year commitment to the Pompe community. We are proud to be building on our heritage in this area and are pleased to see this new treatment now be made available to people with Pompe disease. This approval forms part of our commitment to supporting the rare disease community through the development of potentially life-enhancing treatments that have the potential to improve outcomes and the lives of those living with rare diseases.”
About Pompe and avalglucosidase alfa
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 muscle damage.2 Avalglucosidase alfa 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.1 Avalglucosidase alfa 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 III COMET study.1,5
Avalglucosidase alfa is administered as a monotherapy enzyme replacement therapy, given as an infusion once every two weeks.1
About Sanofi
We 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 potentially 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
Contacts
Sanaz Ayoughi | Communications Lead, UK & Ireland Specialty Care
+44 (0) 7753 717 109 | Sanaz.Ayoughi@sanofi.com
MAT-XU-2205235 (V1.0)
February 2023
References
- Nexviadyme 100 mg Summary of Product Characteristic. Available at: https://mhraproducts4853.blob.core.windows.net/docs/08dabca0c052100988d469f923ef3ca661b561df. Last accessed: February 2023.
- NICE. Avalglucosidase alfa for treating Pompe disease. Technology appraisal guidance [TA821]. Available at: https://www.nice.org.uk/guidance/ta821. Last accessed: February 2023.
- Myozyme 50 mg Summary of Product Characteristic. Available at : https://www.medicines.org.uk/emc/product/263/smpc#gref. Last accessed: February 2023.
- Association for Glycogen Storage Disease (AGSD). Pompe Disease (GSD2). Available at: https://agsd.org.uk/all-about-gsd/gsd-variants/pompe-disease-gsd2/. Last accessed: February 2023.
- Diaz-Manera J, Kishnani P, Kushlaf H, et al. Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial. Lancet Neurol 2021; 20: 1012–26.
- Kishnani P, Kronn D, Brassier A, et al. Safety and efficacy of avalglucosidase alfa in individuals with infantile-onset Pompe disease enrolled in the phase 2, open-label Mini-COMET study: The 6-month primary analysis report. Genetics in Medicine 2022. DOI: https://doi.org/10.1016/j.gim.2022.10.010.
