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Nexviadyme® ▼ (avalglucosidase alfa) (GB only)

Nexviadyme® 

avalglucosidase alfa

This is intended for HCPs practising in Great Britain (England, Scotland and Wales) only.
 

Nexviadyme (avalglucosidase alfa) is indicated for long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid α-glucosidase deficiency).

Profile

Nexviadyme offers all patients with Pompe disease a treatment that is:

A monotherapy
 

With fewer serious treatment-related adverse reactions and IARs than Myozyme in the Phase 3 trial.1,2
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Studied for 8 years
 

A multicentre, multinational, open-label study of Nexviadyme in ERT-experienced and -naïve patients with LOPD (n=24) showed a consistent safety profile and a stable forced vital capacity % yearly change of -0.46 (-0.88, -0.05) and -0.39 (-0.86, 0.08), and a 6 minute-walk test change of -0.27 (-0.72, 0.19) and -1.17 (-1.72, -0.61), in the ERT-experienced and naïve groups respectively.3

Designed for better cellular uptake

Enhancement with 15x M6P, compared with Myozyme (alglucosidase alfa), for improved receptor-mediated uptake into muscle cells and targeting to lysosomes.1
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Efficacious
 

Non-inferior differences versus Myozyme in respiratory (FVC, p=0.0074) and motor function (6MWT, nominal improvement p=0.04).1,2
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Nexviadyme may lower risk of ventilation and
wheelchair use vs. Myozyme4

A disease model created using mathematical simulation modelling in patients with LOPD has shown that Nexviadyme may help reduce the risk of ventilation or wheelchair use compared to Myozyme4


 

Evidence 

Discover the results and evidence supporting Nexviadyme® (avalglucosidase alfa) and how it was studied through several clinical trials.


 

Safety profile

Learn more about the safety and tolerability profile for Nexviadyme® (avalglucosidase alfa) for patients living with Pompe disease.

Mechanism of action

Watch the video below to learn more about how Nexviadyme® is designed to replace the deficient GAA enzyme activity in patients with Pompe disease, which results in lysosomal glycogen accumulation in muscles. It is enhanced with fifteen times M6P, compared with Myozyme, for improved receptor-mediated uptake into muscle cells and targeting to lysosomes.1

Usage

Nexviadyme is a monotherapy that does not require a stabiliser. It is administered via intravenous infusion once every 2 weeks.1 See how to administer Nexviadyme® in the video.

  • The recommended dose is 20 mg/kg of body weight administered once every 2 weeks.
  • For patients with IOPD who experience lack of improvement or insufficient response, while receiving 20 mg/kg, a dose increase to 40 mg/kg should be considered in the absence of safety concerns.
  • In patients who do not tolerate Nexviadyme at 40 mg/kg (e.g., severe hypersensitivity, anaphylactic reactions, or risk of fluid overload), consider decreasing the dose to 20 mg/kg.
  • Nexviadyme treatment should be supervised by a physician experienced in the management of patients with Pompe disease or other inherited metabolic or neuromuscular diseases.
  • Patients may be pre-treated with antihistamines, antipyretics, and/or corticosteroids to prevent or reduce allergic reactions.

Watch how and why Nexviadyme® was designed

Curious to know why Nexviadyme® was developed?

Learn more about how Nexviadyme® was designed

Discover our commitment to rare diseases

Did you know?

Incidence is estimated at 1 in 40,000 people, which would equate to 1,675 Pompe patients in the UK, however there are only 200 estimated diagnosed people with Pompe in the UK.5

Learn more about Nexviadyme®

Nexviadyme® evidence

Explore the results and evidence supporting Nexviadyme® and how it was studied through several clinical trials.

Nexviadyme® safety profile

Find out more about the safety profile of Nexviadyme® (avalglucosidase alfa)

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Nexviadyme® evidence

Explore the results and evidence supporting Nexviadyme® and how it was studied through several clinical trials.

Nexviadyme® safety profile

Find out more about the safety profile of Nexviadyme® (avalglucosidase alfa)


 

Get in touch with us

Do you have questions or need support? We are here to help you.

Clinical trial overviews

Overview of COMET

The phase 3 randomised clinical trial supporting Nexviadyme2
 

Overview of Mini-COMET

The phase 2 clinical trial supporting Nexviadyme's use in Infantile-Onset Pompe Disease (IOPD)6

Overview of Neo1

The phase 1 safety clinical trial supporting Nexviadyme7
 


 

References

  1. Sanofi. Nexviadyme (avalglucosidase alfa). Summary of Product Characteristics. 2024. Available at: https://www.medicines.org.uk/emc/product/14562/smpc#gref.

  2. Diaz-Manera J, 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 Neural. 2021 Dec;20(12):1012-1026.

  3. Byrne BJ, et al. NEO1/NEO-EXT Studies: Long-Term Muscle Quantitative Magnetic Resonance Imaging and Functional Efficacy in Adults With Late-Onset Pompe Disease (LOPD) on Avalglucosidase Alfa Treatment. Poster presented at the 20th Annual WORLDSymposium 2024.

  4. Fournier M, et al. Predicted Time to Wheelchair and Ventilation Events Comparing Avalglucosidase Alfa (AVA) Versus (vs) Alglucosidase Alfa (ALG) Using a Model of Late-Onset Pompe Disease (LOPD). Value in Health ISPOR. 2022 December 25;12(S21)

  5. AGSD, Pompe Disease (GSD2). Website, available online here: https://agsd.org.uk/all-about-gsd/gsd-variants/pompe-disease-gsd2 / [Last accessed February 2024]

  6. Kishnani, et al. Mini-COMET Investigators. 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. Genet Med. 2023 Feb;25(2):100328. doi: 10.1016/j.gim.2022.10.010

  7. Pena LDM, et al. Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalalucosidase alfa (neoGAA) in treatment-naive and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study. Neuromuscul Disord. 2019 Mar;29(3):167-186.

MAT-XU-2203480 (v10.0) Date of preparation: March 2024