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NEXVIADYME (avalglucosidase alfa) - Maladie de Pompe

Opinions on drugs - Posted on Dec 01 2022

Reason for request

First assessment

Key points

Approval of reimbursement for long-term enzyme replacement therapy in patient with Pompe disease (α-glucosidase acid deficiency).

No therapeutic improvement in the care pathway.

Role in therapeutic strategy?

Multidisciplinary therapeutic treatment of the disease is coordinated by a hospital practitioner in concert with a reference or expert centre for metabolic or neuromuscular diseases and in concert with the primary care physician. The specific treatment of Pompe disease is based on enzyme replacement therapy. The objectives of treatment are as follows: improve or stabilise cardiac hypertrophy and function in cases of initial impairment (infantile-onset form); stabilise or slow down degradation of muscle weakness, or prevent or delay loss of deambulation; stabilise or slow down respiratory function degradation, and prevent or delay use of assisted ventilation.

The specific treatment of Pompe disease is based on enzyme replacement therapy. Treatment initiation is subject to multidisciplinary validation by experts from an accredited reference centre.

Role of the medicinal product

NEXVIADYME (avalglucosidase alfa) is the second replacement enzyme to be granted a marketing authorisation for Pompe disease, after MYOZYME (alglucosidase alfa).

In late-onset forms of the disease, considering the findings of the clinical non-inferiority study versus alglucosidase alfa conducted on treatment-naive patients, NEXVIADYME (avalglucosidase alfa) is a first-line treatment, in the same way as MYOZYME (alglucosidase alfa) for the treatment of patients with late-onset forms.

In the absence of robust comparison in infantile-onset forms, it is not possible to rank NEXVIADYME (avalglucosidase alfa) in relation to MYOZYME (alglucosidase alfa), and it represents a new first-line treatment option.

Considering the data from the review of temporary authorisations of use and the mini COMET study, NEXVIADYME (avalglucosidase alfa) treatment may be proposed to patients failing to respond to MYOZYME (alglucosidase alfa) treatment.


Clinical Benefit
Substantial

The Committee deems that the actual clinical benefit of NEXVIADYME (avalglucosidase alfa) is low in the treatment of late-onset forms of Pompe disease.
Low

Clinical Added Value
no clinical added value

Late-onset forms of Pompe disease

Considering:

  • the findings of a study demonstrating the non-inferiority, but not superiority, of NEXVIADYME (avalglucosidase alfa) versus MYOZYME (alglucosidase alfa) in terms of transient benefit on the functional clinically relevant outcome measure, namely FVC assessed after 49 weeks of treatment in hitherto enzyme therapy treatment-naive patients,
  • the uncertainties around the long-term efficacy of NEXVIADYME (avalglucosidase alfa),
  • the safety profile of NEXVIADYME (avalglucosidase alfa) which appears to be favourable,

the Committee deems that NEXVIADYME (avalglucosidase alfa) provides no clinical added value (CAV V) in the therapeutic strategy of late-onset forms of Pompe disease.

Infantile-onset forms of Pompe disease

Considering:

  • the predominantly non-comparative exploratory data from a multi-cohort phase II study, on patients with an infantile-onset form of the disease (which is a more severe form of the disease than the late-onset form) who demonstrated cardiac or muscle parameter stabilisation, lack of degradation, while these patients were previously treated with alglucosidase alfa and exhibited a decline or poor response under this first treatment,
  • the lack of data as to the longer-term progression of heart, muscle and neurological impairments ultimately leading to disability, and on overall survival in patients;
  • the severity of this form of Pompe disease and the partially met medical need,
  • the safety profile of NEXVIADYME (avalglucosidase alfa) which appears to be favourable in the paediatric population,

the Committee deems that NEXVIADYME (avalglucosidase alfa) provides no clinical added value (CAV V) in the therapeutic strategy of infantile-onset forms of Pompe disease.

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