Gene therapy Luxturna® now reimbursed in Quebec for people with previously untreatable inherited vision loss(1)

• Quebec leads the way as the first province to reimburse Luxturna^® (voretigene neparvovec) for previously untreatable inherited retinal disease¹
   
• Approved by Health Canada in 2020, Luxturna^® is a one-time gene therapy that provides healthy copies of the RPE65 gene for retinal cells that are lacking the normally functioning gene²

DORVAL, QC, Oct. 17, 2022 /CNW Telbec/ - Novartis Pharmaceuticals Canada Inc. is pleased to announce Luxturna^® (voretigene neparvovec) is now available to Quebec patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations under the province's public health plan. This announcement follows a positive reimbursement recommendation from the Institut national d'excellence en santé et en services sociaux (INESSS) recognizing the need for treatment options to slow the progression of the disease.

"It has been a long journey for patients waiting for public access to Luxturna^®. We are pleased with the Quebec government's decision and hope that eligible Canadians in other provinces have the same chance to be treated with Luxturna^®," said Doug Earle, President & CEO of Fighting Blindness Canada. "To progressively lose your vision to blindness, especially at a young age, is shattering. Our hope is that other provinces move quickly, because for these Canadians, time is of the essence."

Luxturna^® received Health Canada approval in 2020 and was eagerly anticipated by the vision community as the first pharmacological treatment option for people living with this rare, progressive genetic condition¹. For most of these patients, progressive vision loss and total blindness were part of the life-long burden of the disease.

"This is a pivotal moment for the IRD community and for patients who could benefit from treatment with Luxturna^®," said Andrea Marazzi, Country President, Novartis Canada. "We believe the recognition by INESSS to fund Luxturna^® is vital and gives children and adults with biallelic RPE65 mutation-associated retinal dystrophy a chance for improved vision. We will continue to work towards broad reimbursement through public drug plans across Canada to ensure other Canadians have the opportunity to thrive."

Details regarding the INESSS recommendation are available here. The reimbursement criteria for Luxturna^® (voretigene neparvovec) on Quebec's public health plan are available here.

About RPE65 mutation-associated inherited retinal dystrophy

Mutations in both copies of the RPE65 gene affect approximately 1 in 200,000 people and can lead to blindness^3,4. Early in the disease patients can suffer from night blindness (nyctalopia), loss of light sensitivity, loss of peripheral vision, loss of sharpness or clarity of vision, impaired dark adaptation, and repetitive uncontrolled movements of the eye (nystagmus)⁴. Patients with mutations in both copies of the RPE65 gene may be diagnosed, for instance, with subtypes of either retinitis pigmentosa or Leber congenital amaurosis⁵.

About Novartis in Gene Therapy and Rare Disease

Novartis is at the forefront of cell and gene therapies designed to halt diseases in their tracks or reverse their progress rather than simply manage symptoms. The company is collaborating on the cell and gene therapy frontier to bring this major leap in personalized medicine to patients with a variety of diseases, including genetic disorders and certain deadly cancers. Cell and gene therapies are grounded in careful research that builds on decades of scientific progress. Following key approvals of cell and gene therapies by health authorities, new treatments are being tested in clinical trials around the world.

About Novartis in Canada

Novartis Pharmaceuticals Canada Inc., a leader in the healthcare field, is committed to the discovery, development and marketing of innovative products to improve the well-being of all Canadians. Over the last 5 years, our average annual research and development investment in Canada was $47 million. Located in Dorval, Quebec, Novartis Pharmaceuticals Canada Inc. employs approximately 1,000 people in Canada and is an affiliate of Novartis AG, which provides innovative healthcare solutions that address the evolving needs of patients and societies. The company prides itself on its commitment to diversity and to nurturing an inclusive and inspiring environment. Novartis is recognized as a Great Place to Work®, ranked among the Top 50 Best Workplaces™ in the country and is proudly named on the 2021 Best Workplaces™ for Women in Canada and Best Workplace™ for Mental Wellness lists. For further information, please consult www.novartis.ca. 

About Novartis 

Novartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach more than 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 108,000 people of more than 140 nationalities work at Novartis around the world. Find out more at www.novartis.com.

Luxturna is a registered trademark of Spark Therapeutics Inc., used under license by Novartis Pharmaceuticals Canada Inc.

References

1. Patel U BM, de Léséleuc L, et al. Voretigene Neparvovec: An Emerging Gene Therapy for the Treatment of Inherited Blindness. CADTH Issues in Emerging Health Technologies Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2016. 2018 (169)
2. Novartis Pharmaceuticals Canada Inc. Luxturna^® (voretigene neparvovec) Product Monograph. April 20, 2022
3. Novartis. Data on file. 2018.
4. Astuti GD et al. Comprehensive genotyping reveals RPE65 as the most frequently mutated gene in Leber congenital amaurosis in Denmark. European Journal of Human Genetics 2016; 24: 1071–79.
5. Morimura H et al. Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or Leber congenital amaurosis. Proceedings of the National Academy of Sciences of the USA. 1998; 95: 3088–93.

SOURCE Novartis Pharmaceuticals Canada Inc.

Novartis Media Relations: Katia Kononova, Novartis Pharmaceuticals Communications, + 1 514 633 7873, E-mail: [email protected]