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
Fee Schedule:
Schedule E
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.
Date NOC Issued:
Recommendation Type:
Reimburse with clinical criteria and/or conditions
Final Recommendation:
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)