CELLCEPT (mycophenolate mofetil) - Acute transplant rejection in paediatric patients receiving allogeneic renal, cardiac or hepatic

The clinical benefit of CELLCEPT 250 mg, 500 mg, 1 g/5 mL (mycophenolate mofetil) is substantial in the MA indication extension.

Considering:

• data relating to the prevention of cardiac and hepatic transplant rejection in paediatric patients aged from 1 to 18 years, derived from a review of the literature, albeit with a low level of evidence (non-comparative, observational studies, often single-centre, sometimes old, and including low numbers of patients), suggest that immunosuppression protocols including mycophenolate
  mofetil are effective in terms of reducing the risk of acute transplant rejection, and of enabling the doses of calcineurin inhibitors and steroids to be reduced,
• data relating to the prevention of renal transplant rejection in paediatric patients aged from 1 to 2 years based on a pharmacokinetic study having included 6 children in this age group among the 100 children enrolled, the results of which made it possible to determine appropriate doses for children aged 1 to 2 years and to extrapolate the efficacy in this age group based on data available in older patients,
• the fact that the primary endpoint in the majority of studies is rejection. However, this interim endpoint is debatable since it is not always associated with a reduction in transplant or patient survival,
• the absence of any available robust comparison versus the clinically relevant comparator containing azathioprine in the different paediatric indication extensions; comparative data versus azathioprine having previously been assessed by the Committee in adults only,
• the safety profile of mycophenolate mofetil in the paediatric population, which does not appear to differ from that in older patients, characterised by infections, gastrointestinal disorders, and blood and lymphatic system disorders,
• the lack of available quality of life data,

the Committee deems that CELLCEPT 250 mg, 500 mg, 1 g/5 mL (mycophenolate mofetil) hard capsules, film-coated tablets and powder for oral suspension provides a minor clinical added value (CAV IV) in the current care pathway, which includes the relevant comparators.