Alberta patients with previously untreatable inherited vision loss[1] now have access to gene therapy Luxturna®

• Alberta centre first in Canada to treat patients with Luxturna^® (voretigene neparvovec) through public drug plan funding
  
  
• As a one-time gene therapy, Luxturna^® provides healthy copies of the RPE65 gene for retinal cells that are lacking the normally functioning gene²

DORVAL, QC, Dec. 19, 2022 /CNW/ - Novartis Pharmaceuticals Canada Inc. is pleased to announce that Luxturna^® (voretigene neparvovec) is now available through the Alberta public health plan for patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells. Alberta becomes the second province after Quebec to confirm public drug plan listing for Luxturna^® following the successful conclusion of negotiations with the pan-Canadian Pharmaceutical Alliance (pCPA).

"We congratulate Alberta for making Luxturna^® publicly available and are very excited that eligible patients are already being treated. This is a significant accomplishment for patients and highly anticipated by the patient community across the country. Our hope is that other provinces follow because every day that passes is one day closer to blindness for some Canadians living with these progressive genetic conditions¹," said Dr. Larissa Moniz, Director, Research and Mission Programs, Fighting Blindness Canada.

In 2020 Luxturna^® became the first-ever gene replacement therapy approved by Health Canada. Its arrival marked an important step forward in the treatment of genetic eye disorders. Due to the highly specialized nature of the therapy, Novartis is collaborating with key centres and their multidisciplinary teams to deliver Luxturna^®.

"A momentous decision by an enlightened government comes as an early holiday gift for some Canadians experiencing progressive loss of sight leading to a lifetime of blindness. Alberta is demonstrating true compassion and understanding of the social and financial benefits of allowing individuals to fully participate in all aspects of family, work and society," said Durhane Wong-Rieger, President and CEO of the Canadian Organization for Rare Disorders (CORD). "We are hopeful this leadership will inspire other provinces."

"We applaud the Alberta government for recognizing the potential of Luxturna^® and are proud to deliver this science-based innovation for patients who previously had no available treatment options¹," said Andrea Marazzi, Country President, Novartis Canada. "All eligible patients across the country should have the same opportunity and we will continue to collaborate with decision makers for Luxturna^® to be available in other provinces."

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; Karla Fuentes Arellano, Novartis Pharmaceuticals Communications, + 1 514 633 7873, E-mail: [email protected]