CADTH Canadian Drug Expert Committee recommendation: Cerliponase Alfa (Brineura -- Biomarin Pharmaceutical [Canada] Inc.) indication : for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency
The CADTH Canadian Drug Expert Committee (CDEC) recommends that cerliponase alfa be reimbursed for the treatment of CLN2 disease, also known as TPP1 deficiency, if the following conditions are met: Conditions for Reimbursement. Initiation Criteria. 1. A confirmed diagnosis of CLN2 disease based on T...
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| Format: | eBook |
| Language: | English |
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Ottawa (ON)
CADTH
May 2019, 2019
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| Edition: | Version 1.0. (final) |
| Series: | CADTH common drug review
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| Online Access: | |
| Collection: | National Center for Biotechnology Information - Collection details see MPG.ReNa |
| Summary: | The CADTH Canadian Drug Expert Committee (CDEC) recommends that cerliponase alfa be reimbursed for the treatment of CLN2 disease, also known as TPP1 deficiency, if the following conditions are met: Conditions for Reimbursement. Initiation Criteria. 1. A confirmed diagnosis of CLN2 disease based on TPP1 enzyme activity and CLN2 genotype analysis. 2. Patient who meets all of the following: 2.1. has a minimum score of ≥ 1 in each of the motor and the language domains of the CLN2 Clinical Rating Scale. 2.2. has an aggregate motor-language score of ≥ 3 on the CLN2 Clinical Rating Scale. Discontinuation Criteria: 1. Patients must be assessed every 24 weeks for changes in motor and language function using the CLN2 Clinical Rating Scale. 2. Treatment must be discontinued if: 2.1. there is a reduction of ≥ 2 points in the aggregate motor-language score of the CLN2 Clinical Rating Scale that is maintained over any two consecutive 24-week assessments; or 2.2. the aggregate motor-language score of the CLN2 Clinical Rating Scale reaches zero at two consecutive 24-week assessments |
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| Physical Description: | 1 PDF file (11 pages) |