AUBAGIO (tériflunomide) - SEP récurrentes-rémittentes

Key points

Unfavourable opinion for reimbursement in the treatment of paediatric patients aged 10 years and older with relapsing remitting multiple sclerosis (MS), in view of the available alternatives.

Role in the care pathway?

The early initiation of disease-modifying therapy is recommended following validation of the diagnosis of MS in children. Several therapeutic options are proposed as first-line treatments and have a paediatric MA: beta 1-a interferons (AVONEX and REBIF), beta 1-b interferons (EXTAVIA and BETAFERON) and glatiramer acetate (COPAXONE). REBIF (interferon beta 1-a) is the only one of these medicinal products to have an MA in children aged 2 years and older, with the others only having
an MA for patients aged 12 years and older.

In 2019, the Committee examined the indication extension for GILENYA (fingolimod) as a disease modifying therapy in highly active relapsing remitting multiple sclerosis in patients aged 10 years and older. It considered that GILENYA (fingolimod) was a first or second-line treatment for highly active relapsing remitting multiple sclerosis in the groups of paediatric patients aged 10 years and older defined by the MA, as follows:

• Patients with highly active disease despite a full and adequate course of treatment with at least
  one disease modifying therapy, or
• Patients with rapidly evolving severe relapsing remitting multiple sclerosis defined by 2 or more disabling relapses in one year, and with 1 or more Gadolinium enhancing lesions on brain MRI or a significant increase in T2 lesion load as compared to a previous recent MRI.

The Committee also highlighted that monitoring of patients was required in accordance with the SPC and the risk management plan, due to the safety profile of GILENYA (fingolimod), marked in particular by cardiac rhythm disorders (bradyarrhythmia) after the first dose, infections, seizures and skin cancer.

The proprietary medicinal product TYSABRI (natalizumab) does not have a paediatric MA but is nonetheless cited by the 2019 French national diagnostic and care protocol (PNDS) as a therapeutic option in second-line situations following the failure of immunomodulatory therapies in the event of severe or rapidly evolving disease. It is necessary to contact a reference centre specialising in rare inflammatory brain and spinal cord diseases for the use of this proprietary medicinal product in the
paediatric population.

Role of AUBAGIO (teriflunomide) in the care pathway for the treatment of paediatric patients
aged 10 years and older with relapsing remitting MS:

Considering:

• the non-statistically significant result for the primary endpoint (time to first clinical relapse) in the TERIKIDS comparative study versus placebo, which cannot eliminate the possibility of a loss of opportunity for patients in view of the available clinically relevant comparators,
• the absence of study conducted versus an active comparator, despite this being feasible, in a context in which immunomodulatory substances have been available for several years,
• limited long-term safety data in the paediatric population, particularly in patients beginning this treatment at a very young age,

the Committee considers that AUBAGIO (teriflunomide) has no role in the treatment of paediatric patients aged 10 years and older with relapsing remitting forms of multiple sclerosis (MS).