Novartis and the Pan-Canadian Pharmaceutical Alliance (pCPA) Conclude Negotiations for Luxturna®, a Gene Therapy for Previously Untreatable Inherited Vision Loss(1)

  • Luxturna® (voretigene neparvovec) is a unique gene therapy that provides healthy copies of the RPE65 gene for retinal cells that lack the normally functioning gene1

DORVAL, QC, September 20, 2022 /CNW/ – Novartis Pharmaceuticals Canada Inc. and the pan-Canadian Pharmaceutical Alliance (CPA) have successfully concluded negotiations for Luxturna® (voretigene neparvovec), a single-use gene therapy for the treatment of adult and pediatric patients with vision loss due to hereditary retinal dystrophy caused by confirmed biallelic disease RPE65 mutations.

Hereditary retinal dystrophies (IRDs) are a major cause of early blindness2. RPE65Mediated IRDs are rare, serious, and progressive conditions that eventually lead to severe visual impairment and blindness. Prior to Luxturna approval®there were no pharmacological treatment options available for this form of hereditary blindness2.

“We are delighted to have successfully concluded these negotiations for Luxturna®one of two pioneering targeted gene therapies introduced by Novartis in Canada for patients and families devastated by rare, debilitating or life-threatening genetic diseases. For all Canadians who urgently need treatment with this innovative therapy, this is an important step in gaining access,” said Andrea MarazziPresident of the country, Novartis Canada. “We will continue to work with provincial and territorial authorities to ensure that patients whose vision is impaired due to a mutation in both copies of RPE65 the gene can gain access to Luxturna® through public drug plans as soon as possible. »

“This type of inherited eye disease affects children and young adults and creates a significant impact on the whole family,” said Doug EarlePresident and CEO of Fighting Blindness Canada.

“For the majority, this can lead to complete blindness as cells in the retina, the light-sensitive tissue of the eye, gradually stop functioning, work less efficiently, or die.2. Having access to treatment that can help restore sight can be life changing for a child or young adult and their family. We encourage provinces to recognize the Luxturna hope® represents and prioritizes access, as there are no other approved treatments available to these Canadians. »

About RPE65 hereditary retinal dystrophy associated with a mutation
Mutations in both copies of the RPE65 affects around 1 in 200,000 people and can lead to blindness3.4. Early in the disease, patients may experience night blindness (nyctalopia), loss of sensitivity to light, loss of peripheral vision, loss of sharpness or clarity of vision, poor adaptation to darkness and uncontrolled repetitive eye movements (nystagmus)4. Patients with mutations in both copies of the RPE65 can be diagnosed, for example, with subtypes of retinitis pigmentosa or Leber congenital amaurosis5.

About Novartis in Gene Therapy and Rare Diseases
Novartis is at the forefront of cell and gene therapies designed to stop diseases in their tracks or reverse their progression rather than simply manage symptoms. The company is collaborating at the frontier of cell and gene therapy to bring this major advancement in personalized medicine to patients with various diseases, including genetic disorders and certain life-threatening cancers. Cell and gene therapies are based on careful research that builds on decades of scientific advancements. Following key cell and gene therapy approvals 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 healthcare, is committed to discovering, developing and bringing to market innovative products to improve the well-being of all Canadians. Over the past 5 years, our average annual investment in research and development in Canada has been $47 million. Situated in Dorval, QuebecNovartis Pharmaceuticals Canada Inc. employs approximately 1,000 people in Canada and is a subsidiary of Novartis AG, which provides innovative healthcare solutions that meet the evolving needs of patients and societies. The company prides itself on its commitment to diversity and fostering an inclusive and inspiring environment. Novartis is recognized as a Great Place to Work®, ranked among the nation’s 50 Best Workplaces™ and proudly named on the 2021 Best Workplaces™ for Women in Canada and the Best Workplace™ for Mental Wellness lists. For more information, please see

About Novartis
Novartis is reinventing medicine to improve and extend people’s lives. As a world leader in medicines, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest for new medicines, we consistently rank among the top companies in the world that invest in research and development. Novartis products reach more than 800 million people worldwide and we are finding innovative ways to expand access to our latest treatments. Approximately 108,000 people of more than 140 nationalities work at Novartis worldwide. Learn more about

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


  1. Novartis Pharmaceuticals Canada Inc. Luxturna® (voretigene neparvovec) Product Monograph. April 20, 2022
  2. Patel U BM, de Léséleuc L, et al. Voretigene Neparvovec: an emerging gene therapy for the treatment of hereditary blindness. CADTH issues in new health technologies Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2016. 2018 (169)
  3. Novartis. Data on file. 2018.
  4. Astuti GD et al. Comprehensive genotyping reveals that RPE65 is 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. RPE65 gene mutations in patients with autosomal recessive retinitis pigmentosa or Leber congenital amaurosis. Proceedings of the National Academy of Sciences UNITED STATES. 1998; 95: 3088–93.

SOURCE Novartis Pharmaceuticals Canada Inc.

For further information: Novartis Media Relations, Daphne Weatherby, Novartis Pharmaceuticals Communications, +1 514 633 7873, Email: [email protected]