Voretigene neparvovec (Luxturna) is a gene therapy indicated for the treatment of people experiencing vision loss due to inherited retinal dystrophy caused by an RPE65 gene mutation.
The treatment is approved by Health Canada for adults and children between the ages of four and 65. It is a one-time gene replacement therapy delivered by subretinal injection.
The process to deliver voretigene neparvovec requires specialized expertise and equipment that is only available at select Ontario hospitals.
Participating Ontario Hospitals
- Sunnybrook Health Sciences Centre
- The Hospital for Sick Children
What You Need to Know Before You Refer a Patient
- To refer a patient, you must be an ophthalmologist licensed to practice in Ontario.
- The patient must have a valid Ontario health card.
If you have any questions about funding or enrolment, please email Provincial Drug Reimbursement Programs
Please do not send Ontario Health personal health information.
Enrol a Patient
- Contact The Hospital for Sick Children (Dr. Elise Heon).
- Confirm that your patient meets all of the eligibility criteria listed in the following section.
- The Hospital for Sick Children will complete a clinical assessment, confirm the patient’s eligibility and determine which hospital will deliver the treatment.
- The treating facility will then enrol the patient by completing the enrolment form and submitting it via our online system, eClaims.
Eligibility criteria
The patient must meet all of the following criteria:
Biallelic retinal pigment epithelium 65 kDa protein (RPE65) mutations, as confirmed by an accredited laboratory using validated assay methods and discussed with the medical/clinical geneticists
Possess sufficient viable retinal cells in each eye to be treated, as determined by an inherited retinal disease specialist through non-invasive means such as optical coherence tomography (OCT) and/or ophthalmoscopy and by at least 1 of the following:
- Retinal thickness within the posterior pole of greater than 100 µm as shown on OCT, or
- 3 or more disc areas of retina without atrophy or pigmentary degeneration within the posterior pole, or
- a remaining visual field within 30 degrees of fixation as measured by III4e isopter or equivalent, or
- measurable full-field stimulus testing (FST)
At least 4 years and under 65 years of age
Visual acuity worse than 20/60 (both eyes) and/or visual field less than 20 degrees and/or measurable FST
Has not received a previous treatment course of voretigene neparvovec
Related Document
High Cost Therapy Funding Program Policy
Learn the processes for the application and reimbursement of voretigene neparvovec in Ontario.