Health Canada approves Zolgensma®, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA)

•  SMA is a rare genetic disease that leads to progressive muscle weakness, paralysis and, when left untreated in its most severe form, permanent ventilation or death for most patients by age 2^2,3
• Zolgensma (onasemnogene abeparvovec) is approved for the treatment of pediatric SMA patients with 3 or fewer copies of the SMN2 gene, or infantile-onset SMA¹
• Zolgensma is designed to address the genetic root cause of SMA by replacing the missing or defective SMN1 gene to halt disease progression¹

DORVAL, QC, Dec. 16, 2020 /CNW/ - Novartis Pharmaceuticals Canada Inc. is pleased to announce that Health Canada has approved Zolgensma^® (onasemnogene abeparvovec) an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of pediatric patients with 5q spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene and 3 or fewer copies of SMN2 gene; or infantile-onset SMA¹.

Zolgensma MOA B-Roll

Understanding-SMA infographic (CNW Group/Novartis Pharmaceuticals Canada Inc.)

Zolgensma is a gene therapy designed to address the genetic root cause of SMA by replacing the missing or defective SMN1 gene¹. It is administered during an intravenous (IV) infusion, delivering a new working copy of the SMN1 gene into a patient's cells, halting disease progression and restoring production of SMN protein¹.

"SMA can be a devastating diagnosis for families to receive. Without treatment, many children would not be able to meet important developmental milestones like lifting their head, sitting or walking. Even breathing and swallowing can become difficult in the severe, infant-onset form of this disease," said Dr. Hugh McMillan, Pediatric Neurologist at the Children's Hospital of Eastern Ontario in Ottawa. "The approval of Zolgensma in Canada offers children an opportunity to maximize their developmental potential from this one-time therapy. The decision to treat based upon weight may allow children diagnosed slightly later to also benefit from this therapy."

"When I first started diagnosing SMA, I couldn't have imagined that we would see such scientific advancements," said Dr. Nicolas Chrestian, Chief of Paediatric Neurology, specialized in neuromuscular disorders at Centre Hospitalier Mère Enfant Soleil, Université Laval in Québec City. "Zolgensma offers, in a single dose, the possibility of halting the progression of this degenerative condition that can rob children of regular developmental milestones."

In Canada each year, approximately one in 10,000 babies are born with SMA, a rare, genetic neuromuscular disease caused by a defective or missing SMN1 gene³. Without a functional SMN1 gene, infants with SMA lose the motor neurons responsible for muscle functions such as breathing, swallowing, speaking and walking². Left untreated, muscles become progressively weaker^2,3. In the most severe form, this eventually leads to paralysis and ultimately permanent ventilation or death by age 2 in more than 90% of cases⁴.

"The SMA community is thrilled to have another treatment option to offer hope to families grappling with an SMA diagnosis. The approval of Zolgensma couldn't come soon enough. We will continue to advocate until everyone who needs access to treatment can benefit from innovations like this," said Susi Vander Wyk, Executive Director, CureSMA Canada.

"Today's announcement about the Canadian approval of Zolgensma is a significant milestone in our journey to reimagine medicine by changing the treatment paradigm for children with SMA." said Andrea Marazzi, Country Head, Novartis Pharmaceuticals Canada. "Our commitment to the SMA community truly comes to life when those that could benefit most from Zolgensma can access it. This is why we continue to work collaboratively with the pan-Canadian Pharmaceutical Alliance, provinces and territories to make this happen as quickly as possible."

The efficacy and safety data supporting the approval of Zolgensma in treating pediatric patients with SMA are derived from completed and ongoing open-label, single-arm, clinical trials in patients with infantile-onset SMA and 2 copies of SMN2 gene; and presymptomatic genetically diagnosed SMA and 2 or 3 copies of SMN2 gene¹.

Zolgensma is the only gene therapy approved by Health Canada for the treatment of SMA¹. Thirteen treatment sites have been identified in leading healthcare institutions with SMA expertise. The sites are located in: Vancouver, BC; Edmonton, AB; Calgary, AB; Saskatoon, SK; Winnipeg, MB; London, ON; Hamilton, ON; Toronto, ON; Ottawa, ON; Montreal, QC; Quebec City, QC; Halifax, NS.

About Spinal Muscular Atrophy 
SMA is the leading cause of genetic infant death². Loss of motor neurons cannot be reversed, so SMA patients with symptoms at the time of treatment will likely require some supportive respiratory, nutritional and/or musculoskeletal care to maximize functional abilities⁵. This is why it is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible to halt irreversible motor neuron loss and disease progression⁶. Early diagnosis is especially critical in the most severe form, where motor neuron degeneration starts before birth and escalates quickly⁵. Newborn screening for SMA is currently being implemented in Ontario and piloted in Alberta^7,8.

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. In 2019, the company invested $51.8 million in research and development in Canada. Located in Dorval, Quebec, Novartis Pharmaceuticals Canada Inc. employs approximately 1,500 people in Canada and is an affiliate of Novartis AG, which provides innovative healthcare solutions that address the evolving needs of patients and societies. For further information, please consult www.novartis.ca.   

About Novartis globally
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 nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 110,000 people of more than 140 nationalities work at Novartis around the world. Find out more at https://www.novartis.com.

Zolgensma is a registered trademark of Novartis Gene Therapies.

Novartis Gene Therapies has an exclusive, worldwide license with Nationwide Children's Hospital to both the intravenous and intrathecal delivery of AAV9 gene therapy for the treatment of all types of SMA; has an exclusive, worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for the in vivo gene therapy treatment of SMA in humans; an exclusive, worldwide licensing agreement with Généthon for in vivo delivery of AAV9 vector into the central nervous system for the treatment of SMA; and a non-exclusive, worldwide license agreement with AskBio for the use of its self-complementary DNA technology for the treatment of SMA.

References

1. Novartis Pharmaceuticals Canada Inc. Zolgensma^® (onasemnogene abeparvovec) Product Monograph. December 15, 2020.
2. Anderton RS and Mastaglia RL. Advances and challenges in developing a therapy for spinal muscular atrophy. Expert Rev Neurother. 2015;15(8)895-908.
3. National Organization for Rare Disorders (NORD). Spinal Muscular Atrophy. Available at http://rarediseases.org/rarediseases/spinal-muscular-atrophy/. Last accessed November 16, 2020
4. Finkel RS, et al. Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurology. 2014;83(9):810-817.
5. Wang CH, et al. Consensus Statement for Standard of Care in Spinal Muscular Atrophy. J Child Neurol. 2007;22(8):1027-1049.
6. Govoni A et al. Time Is Motor Neuron: Therapeutic Window and Its Correlation with Pathogenetic Mechanisms in Spinal Muscular Atrophy. Mol Neurobiol. 2018 Aug;55(8):6307-6318.
7. Newborn Screening Ontario. Spinal Muscular Atrophy (SMA). Available at https://www.newbornscreening.on.ca/en/disease/spinal-muscular-atrophy-sma. Last accessed November 2, 2020.
8. Alberta Children's Hospital. In search of hope for kids with Spinal Muscular Atrophy. Available at http://www.childrenshospital.ab.ca/site/PageNavigator/news/2019_SMA_V4.html. Last accessed November 2, 2020.

SOURCE Novartis Pharmaceuticals Canada Inc.

Novartis Media Relations, Julie Schneiderman, [phone], E-mail: [email protected]