Generic Name: onasemnogene abeparvovec
Brand Name: Zolgensma
Manufacturer: Novartis Pharmaceuticals Canada Inc.
Indications: Spinal muscular atrophy (SMA), pediatrics
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: New
Project Status: Active
Companion Diagnostics: No
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 |
| Draft CADTH review report(s) provided to sponsor for comment | September 25, 2020 |
| Deadline for sponsors comments | October 06, 2020 |
| CADTH responses on draft review report(s) provided to sponsor | November 06, 2020 |
| Expert committee meeting (initial) | November 18, 2020 |
| Draft recommendation issued | November 30, 2020 To December 02, 2020 |