National Centre for Pharmacoeconomics

National Centre for

NCPE Ireland

Sebelipase alfa (Kanuma®)

Sebelipase alfa is indicated for long-term enzyme replacement therapy (ERT) in patients of all ages with lysosomal acid lipase (LAL) deficiency.

NCPE Assessment Process Complete
Rapid review received  08/02/2017
Rapid review completed  13/03/2017
Rapid Review outcome A Full pharmacoeconomic assessment is recommended
Full pharmacoeconomic assessment commissioned by HSE  30/03/2017
Pre-submission consultation with Applicant  22/08/2017
Submission received from Applicant  13/11/2017
Preliminary review sent to Applicant 16/03/2018
NCPE assessment re-commenced 11/04/2018
Factual accuracy sent to applicant 17/05/2018
NCPE assessment re-commenced 05/06/2018
NCPE assessment completed 08/06/2018
NCPE assessment outcome The NCPE recommends for the infantile presentation, that sebelipase alfa (Kanuma®) not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatments. For the paediatric adult presentation, robust evidence of additional clinical benefit of sebelipase alfa has not been demonstrated. Therefore the NCPE recommends that sebelipase alfa not be considered for reimbursement  These recommendations should be considered while also having regard to the criteria specified in the Health (Pricing and Supply of Medical Goods) Act 2013.


The HSE approved reimbursement following confidential price negotiations. July 2019.