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TECFIDERA (diméthyle fumarate) - sclérose en plaques de forme rémittente-récurrente (SEP-RR)

Opinions on drugs - Posted on Dec 22 2022

Reason for request

New indication

Key points

Approval of reimbursement for the treatment of children aged 13 years and over with remitting-relapsing multiple sclerosis (RR-MS).

Therapeutic improvement?

No therapeutic improvement.

Role in therapeutic strategy?

The therapeutic management of childhood MS is multimodal and is based on pharmacological measures accompanied by non-medicinal measures (functional rehabilitation, speech therapist, orthoptist, psychologist, etc., along with social and educational measures). It has short- and long-term objectives: in the short term, the objective is to prevent and manage episodes, reduce their frequency and preserve the child’s integration and psychosocial and emotional development. In the long term, the objective is to limit impairment, maintain follow-up (compliance and tolerance), and limit the cumulative side-effects of medicinal products.

Early set-up of a basic treatment is recommended once the diagnosis of childhood MS is confirmed. Several therapeutic options are proposed as a first-line approach and have been granted a paediatric marketing authorisation: interferon beta 1-a drugs (AVONEX and REBIF), interferon beta 1-b drugs (EXTAVIA and BETAFERON) and glatiramer acetate (COPAXONE). Of these proprietary medicinal products, only REBIF (interferon beta 1-a) has been granted a marketing authorisation for children aged 2 years and over, the others only for patients aged over 12 years.

In 2019, the Committee assessed the extension of the indication of the proprietary medicinal products GILENYA (fingolimod) as a basic treatment of very active forms of RR-MS in patients aged 10 years and over. It considered GILENYA (fingolimod) as a first- or second-line treatment of very active forms of RR-MS for the paediatric patient cohorts aged 10 years and over defined by the marketing authorisation, as follows:

  • patients with a very active form of the disease despite well-managed full treatment with at least one basic MS treatment or,
  • patients with rapidly progressive severe RR-MS, defined by 2 incapacitating episodes in the course of a year associated with 1 or more raised lesion(s) after Gadolinium injection on a brain MRI or a significant increase in the lesion load in T2 compared to a recent prior MRI.

The Committee also pointed out that patient monitoring was required, in accordance with the SPC and RMP, on account of the safety profile of GILENYA (fingolimod), particularly marked by heart rhythm disorders, convulsions, and skin cancer.

The proprietary medicinal product TYSABRI (natalizumab) has not been granted a paediatric marketing authorisation but is nonetheless listed by the 2019 PNDS as a second-line therapeutic option after failure to respond to immunomodulatory drugs in cases of rapidly progressing severe disease. A reference centre for rare inflammatory brain and bone marrow diseases should be contacted for the use of this proprietary medicinal product in the paediatric population.

Role of TECFIDERA (dimethyl fumarate) in the therapeutic strategy for the treatment of children aged 13 years and over with RR-MS

TECFIDERA (dimethyl fumarate) is an alternative to the other first-line basic treatments indicated for RR-M in adolescents aged 13 years and over.

As for adults, it is necessary to perform a lymphocyte count prior to treatment initiation and monitor lymphopenia onset throughout treatment. Uncertainties remain around potential side-effects on growth and fertility in children, particularly prepubescent children.

The summary of product characteristics (SPC) and the Risk Management Plan (RMP) must be adhered to.

Clinical Benefit


The Committee deems that the clinical benefit of TECFIDERA (dimethyl fumarate) is significant in the paediatric indication extension for the treatment of children aged 13 years and over with RR-MS.

Clinical Added Value

no clinical added value


  • the efficacy data from a study versus interferon beta-1a (AVONEX) on the proportion of patients with no new or enlarged hyperintense lesion in T2 in a brain MRI in adolescents mostly aged from 13 to 18 years (90% of the patients included) with RR-MS, the interpretation of which is merely descriptive insofar as no statistical hypothesis has been formulated, or the number of subjects needed calculated,
  • the lack of robust quality-of-life data,

and also considering:

  • the efficacy data assessed during initial inclusion in adults,
  • the overall similar safety profile in the adolescent population to that of the adult population, despite the limited follow-up (study up to 96 weeks), with in particular the risk of onset of lymphopenia, and uncertainties around growth and fertility in children in particular prepubescent children,
  • the need for dosage formulations suitable for the adolescent population in a context where the alternatives available are administered by the injectable route,

the Committee deems that TECFIDERA (dimethyl fumarate) provides no clinical added value (CAV V) in the therapeutic strategy for the treatment of children aged 13 years and over with RR-MS.




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