Revised SPC: Kanuma (sebelipase alfa) 2mg/ml concentrate solution

SPC has been revised with further dosing information and safety data based on 125 patients exposed to sebelipase alfa at doses ranging from 0.35 mg/kg once every other week to 7.5 mg/kg once weekly in clinical studies with a treatment duration from 1 day to 60.5 months.

SPS commentary:

The SPC makes the following new dose recommendations:

  • In infants (< 6 months of age), dose escalation to 3 mg/kg should be considered in case of suboptimal clinical response after a minimum of 4 infusions; a further dose escalation up to 5 mg/kg should be considered in case of suboptimal clinical response after a minimum of additional 4 infusions. dose adjustments, as a reduction of the dose or an extension of the dose interval, can be made on an individual basis based on achievement and maintenance of therapeutic goals. Clinical studies evaluated doses ranging from 1 to 5 mg/kg once weekly, with one patient receiving a higher dose of 7.5 mg/kg once weekly. Doses higher than 7.5 mg/kg have not been studied.
  • In children and adults, dose escalation to 3 mg/kg once every other week should be considered based on clinical response.

The following very common adverse effects have been added to SPC: hypersensitivity, anaphylactic reaction, vomiting, pyrexia, presence of drug specific antibody, hypotension, abdominal pain, diarrhoea and fatigue.

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