Health Canada Approves First Sight-Restoring Gene Therapy for Inherited Eye Diseases

Thursday, October 15 2020 | 10 h 23 min | News, Press Release

On October 14, 2020, Health Canada announced their approval of the first sight-restoring innovative treatment voretigene neparvovec (Brand name: Luxturna). This announcement is the first targeted gene therapy approved in Canada for any disease. This treatment is specifically for individuals with the inherited blinding eye diseases retinitis pigmentosa (RP) or Leber congenital amaurosis (LCA) resulting from mutations in the RPE65 gene that leads to vision loss and eventually blindness.

“Lives will be transformed in Canada because of approval of this research-delivered groundbreaking treatment. For the first time, it means we have a treatment option – bringing hope to families affected by genetic mutations causing blindness and to everyone in the vision loss community.” says Doug Earle, President & CEO, Fighting Blindness Canada.  

For families like the Gandhi’s, the approval of this treatment has been years in the waiting. Sophia Gandhi, mother of Zara, 10 years old born with blindness due to LCA from the RPE65 gene mutation says, “Zara was diagnosed with blindness at 6 months old. We had great doctors, but treatment options were not available to help improve Zara’s limited sight. When we learned about Luxturna many years ago, we knew one day it must and would come to Canada (…).”

Dr. Elise Héon a clinician-scientist in the field of ocular genetics with The Hospital for Sick Children in Toronto, and Zara’s eye doctor says, “This milestone approval of Luxturna is a significant step forward in the treatment of inherited blinding eye diseases. Until now, patients like Zara had no treatment options and the progression towards complete blindness was inevitable. These families now have hope with the promise of a one-time treatment option that can improve or restore vision, especially night vision.” The Hospital for Sick Children will be one of the sites for this treatment in Canada.

While this treatment has been approved by Health Canada, families are awaiting recommendations from Canadian Agency for Drugs and Technologies In Health (CADTH) and the Institut national d’excellence en santé et en services sociaux (INESSS) for coverage in provincial drug benefit programs later this fall.

Earle adds, “People with vision loss due to the RPE65 mutation for the first time in history have a chance for improved vision. And now, it’s the provincial governments turn. Health Canada’s approval of the treatment will not mean much if Canadian families can’t afford to access it. We are calling on the provinces to fund this groundbreaking treatment for Canadian families in need.”

“Fighting Blindness Canada urges all eye health practitioners to encourage their patients and families to continue supporting the science by getting their genetic testing done. Genetic testing will identify if they can be treated now and help advance research of all inherited blinding eye diseases.” says Doug Earle. “Please visit FightingBlindness.ca to obtain a step by step guide of how to get genetic testing done in your province.”

Click HERE for the full press release.