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onasemnogene abeparvovec

Last Updated: November 27, 2020
Result type: Reports
Project Number: SG0649-000
Product Line: Reimbursement Review

Generic Name: onasemnogene abeparvovec

Brand Name: Zolgensma

Manufacturer: Novartis Pharmaceuticals Canada Inc.

Therapeutic Area: Spinal muscular atrophy (SMA), pediatrics

Indications: ​Zolgensma is an adeno-associated virus (AVV) vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

Manufacturer Requested Reimbursement Criteria1: Zolgensma is an adeno-associated virus (AVV) vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene

Submission Type: Initial

Project Status: Active

Companion Diagnostics: No

Fee Schedule: Schedule E

  1. The requested reimbursement criteria are provided by the applicant and do not necessarily reflect the views of CADTH. Reimbursement criteria from CADTH will be documented in the final recommendation, if applicable.

Key Milestones2

Call for patient input openMay 26, 2020
Call for patient input closedJuly 15, 2020
Clarification:

- Patient input submission received from Cure SMA Canada and Muscular Dystrophy Canada (MDC)

Submission receivedJune 25, 2020
Submission acceptedJuly 10, 2020
Review initiatedJuly 13, 2020
Clarification:

- Selected for CADTH/INESSS Joint Clinician Engagement

Draft CADTH review report(s) provided to sponsor for commentSeptember 29, 2020
Deadline for sponsors commentsOctober 08, 2020
CADTH responses on draft review report(s) provided to sponsorNovember 06, 2020
Expert committee meeting (initial)November 18, 2020
Draft recommendation issued to sponsor-
Clarification:

- CDEC recommendation held pending receipt of NOC and evaluation of the finalized submission requirements.