Sebelipase alfa (Kanuma®)

Assessment Status Assessment Process Complete
Drug Sebelipase alfa
Brand Kanuma®
Indication Is indicated for long-term enzyme replacement therapy (ERT) in patients of all ages with lysosomal acid lipase (LAL) deficiency.
Assessment Process
Rapid review commissioned 08/02/2017
Rapid review completed 13/03/2017
Rapid review outcome Full Pharmacoeconomic Assessment Recommended
Full pharmacoeconomic assessment commissioned by HSE 30/03/2017
Pre-submission consultation with Applicant 22/08/2017
Full 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.

Technical Summary

The HSE approved reimbursement following confidential price negotiations July 2019.