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
NOC Status at Filing: Pre NOC
Project Status: Active
Companion Diagnostics: No
Date Recommendation Issued: March 24, 2021
Recommendation Type: Reimburse with clinical criteria and/or conditions
Fee Schedule: Schedule E
- 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 open | May 26, 2020 |
| Call for patient input closed | July 15, 2020 |
| Clarification:
- Patient input submission received from Cure SMA Canada and Muscular Dystrophy Canada (MDC) |
|
| Submission received | June 25, 2020 |
| Submission accepted | July 10, 2020 |
| Review initiated | July 13, 2020 |
| Clarification:
- Selected for CADTH/INESSS Joint Clinician Engagement |
|
| Draft CADTH review report(s) provided to sponsor for comment | September 29, 2020 |
| Deadline for sponsors comments | October 08, 2020 |
| CADTH responses on draft review report(s) provided to sponsor | November 06, 2020 |
| Expert committee meeting (initial) | November 18, 2020 |
| Draft recommendation issued to sponsor | December 17, 2020 |
| End of embargo period | February 05, 2021 |
| Clarification:
- Request for extension to feedback period received from the sponsor - Feedback extension request granted - Reconsideration requested - Request for Clarification received from DRR Participating Drug Plans |
|
| Expert committee meeting | March 17, 2021 |
| Final recommendation issued to sponsor and drug plans | March 24, 2021 |
| Final recommendation posted | March 26, 2021 |
| Deadline for sponsor to submit redaction requests on draft CADTH review report(s) | April 08, 2021 |
| CADTH review report(s) posted | |