Product Details

Zolgensma

Onasemnogene Abeparvovec
20,000,000,000,000 VG/mL
Solution for Infusion
Single-Use Vial Combipack

DIN/PIN/NPN

02509695

Manufacturer

Novartis Pharma Canada Inc.

Formulary Listing Date

2021-10-29  

Unit Price

2,910,500.0000

Amount MOH Pays

2,910,500.0000

Coverage Status

Exceptional Access Program Product

ODB Formulary Therapeutic Classification

Therapeutic Note

NO

ATC Code

M09AX09

Interchangeable Products

NO  

LU Clinical Criteria

NO  

EAP Criteria

Therapeutic Class Reimbursement Criteria
Metabolic/Genetic Modifiers

Onasemnogene abeparvovec

  • Brand(s): Zolgensma
  • Dosage Form/Strength: 2 x 1013 vector genomes (VG)/mL solution for infusion
  • Effective date: October 29, 2021

For the treatment of spinal muscular atrophy (SMA) in individuals meeting all the following criteria: 

  1. Diagnosis of SMA is confirmed by genetic documentation of 5q spinal muscular atrophy with biallelic mutations in the survival motor neuron 1 (SMN1) gene; AND 

  1. Patient is 180 days of age or younger at the time that the treatment is administered; AND 

  1. Patient is pre-symptomatic or symptomatic with one to three copies of the survival motor neuron 2 (SMN2) gene; AND 

  1. Patient does not require permanent ventilatory support (invasive or non-invasive)* or a permanent feeding tube; AND 

  1. Patient must be under the care of a specialist with experience in the diagnosis and management of spinal muscular atrophy. 

*Permanent ventilatory support is defined as the need for a tracheostomy or requirement of 16 hours or more of respiratory assistance per day (via non-invasive ventilatory support) for 14 or more consecutive days in the absence of an acute reversible illness excluding perioperative ventilation. 

Exclusion Criteria: 

  1. Reimbursement is limited to one lifetime administration of onasemnogene abeparvovec. Patients who have received a prior dose of onasemnogene abeparvovec accessed by any mechanism (e.g., private insurance plan, clinical trial, compassionate access) will not be funded. 

  1. Patients with 4 (four) or more copies of SMN2 gene will not be funded. 

Notes: 

  1. Patients must test for the presence of anti-AAV9 antibodies and provide the report of antibody titers. It should be noted that patients with titers above 1:50 will not be funded. Repeat testing results will be considered for newborns suspected of having acquired interim antibodies from maternal transmission. 

  1. No further treatment with nusinersen or other medications indicated for the treatment of SMA will be considered after the patient has received a dose of onasemnogene abeparvovec. 

Dosage recommended: A maximum single lifetime dose of 1.1 x 1014 vector genomes (vg)/kg 

Renewal of therapy is not considered.

EAP Drug Request Form:

Standard Form for EAP Drug Requests

Product Monograph

View Monograph