- SOD1-ALS affects approximately 2% of people living with ALS worldwide1
- If approved, tofersen would be the world’s first treatment to target a genetic cause of ALS
- EMA acceptance follows FDA acceptance of tofersen NDA earlier this year
CARLSBAD, Calif., December 5, 2022 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MA) for review of tofersen, an investigational drug for the treatment of superoxide dismutase 1 ( SOD1) amyotrophic lateral sclerosis (ALS). SOD1-ALS is a progressive and uniformly fatal disease that affects fewer than 1,000 people across Europe.2
The EMA is the second regulatory agency to accept review of a marketing application for tofersen after acceptance by the US Food and Drug Administration (FDA) earlier this year. The FDA has set a PDUFA date for tofersen from April 25, 2023. There is currently no targeted treatment for SOD1-ALS.
“The EMA’s acceptance of tofersen MAA is an important development for individuals and families battling SOD1-ALS,” said C. Frank Bennett, Ph.D., Executive Vice President, Chief Scientific Officer and Head of Franchise for Neurological Programs at Ionis. “If approved, tofersen will be the first treatment in the world that targets a genetic cause of ALS. Tofersen also reinforces Ionis’ platform strategy to target other neurological diseases, including other forms of ALS.
The marketing authorization includes the results of the phase 3 VALOR study, its open-label extension study (OLE), a phase 1 study in healthy volunteers and a phase 1/2 study evaluating increasing dose levels. Also included are the most recent 12-month integrated results from the VALOR and OLE study which were recently published in the New England Journal of Medicine.
Biogen has announced that it will maintain its early access program for tofersen, which is now available in 34 countries. Biogen also announced that it will continue to actively work with other regulators around the world and provide updates as needed.
Tofersen is an antisense drug being evaluated for the potential treatment of SOD1-ALS. Tofersen binds to SOD1 mRNA, allowing it to be degraded by RNase-H with the aim of reducing the synthesis of SOD1 protein production. In addition to the ongoing open-label expansion of VALOR, tofersen is being studied in the Phase 3 ATLAS study designed to assess whether tofersen can delay clinical onset when initiated in presymptomatic individuals with a SOD1 genetic mutation and biomarkers of disease activity. Biogen has licensed tofersen from Ionis under a collaborative development and licensing agreement.
About Amyotrophic Lateral Sclerosis and SOD1-ALS
Amyotrophic lateral sclerosis (ALS) is a rare, progressive and fatal neurodegenerative disease that results in the loss of motor neurons in the brain and spinal cord responsible for controlling voluntary muscle movement. People with ALS suffer from muscle weakness and atrophy, which causes them to lose their independence as they gradually lose the ability to move, talk, eat and eventually breathe. The average life expectancy for people with ALS is three to five years from the onset of symptoms. Patients with certain SOD1 mutations have an even shorter life expectancy.
Several genes have been implicated in ALS. Genetic testing helps determine if a person’s ALS is associated with a genetic mutation, even in people with no family history of the disease. Currently, there are no genetically targeted treatment options for ALS. Mutations in the SOD1 gene are responsible for approximately 2% of the estimated 168,000 people with ALS worldwide (SOD1-ALS).
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been the leader in RNA-targeted therapy, opening new markets and changing standards of care with its novel antisense technology. Ionis currently has three marketed drugs and a leading late-stage pipeline highlighted by industry-leading cardiovascular and neurological franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision to become a leading, fully integrated biotechnology company.
To learn more about Ionis, visit www.ionispharma.com and follow us on Twitter @ionispharma.
Ionis Forward-Looking Statements
This press release contains forward-looking statements regarding Ionis’ business and the therapeutic and commercial potential of Ionis’ technologies, tofersen and other products in development. Any statement describing Ionis’ objectives, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. These statements are subject to certain risks and uncertainties, including those related to the impact that COVID-19 may have on our business, and including, but not limited to, those related to our commercial products and pharmaceuticals. our pipeline, and in particular those inherent in the process of discovering, developing and commercializing safe and effective drugs for human therapeutic use, and in the effort to build a business around these drugs. Ionis’ forward-looking statements also involve assumptions which, if they never materialize or prove to be incorrect, could cause its results to differ materially from those expressed or implied by such forward-looking statements.
Although Ionis’ forward-looking statements reflect the good faith judgment of its management, such statements are based solely on facts and factors currently known to Ionis. Accordingly, you are cautioned not to rely on these forward-looking statements. These and other risks relating to Ionis’ programs are further described in Ionis’ Annual Report on Form 10-K for the fiscal year ended. December 31, 2021, and the most recent quarterly filing of Form 10-Q, which are filed with the Securities and Exchange Commission. Copies of these and other documents are available from the Company.
In this press release, unless the context otherwise requires, “Ionis”, “Company”, “we”, “us” and “our” refer to Ionis Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is a trademark of Ionis Pharmaceuticals, Inc.
1 Brown CA, Lally C, Kupelian V, Flanders WD. Estimated prevalence and incidence of amyotrophic lateral sclerosis and genetic variants SOD1 and C9orf72. Neuroepidemiology. 2021;55(5):342-353. doi: 10.1159/000516752. Published online July 9, 2021.
2 Brown CA, Lally C, Kupelian V, Flanders WD. Estimated prevalence and incidence of amyotrophic lateral sclerosis and genetic variants SOD1 and C9orf72. Neuroepidemiology. 2021;55(5):342-353. doi: 10.1159/000516752. Published online July 9, 2021.
SOURCEIonis Pharmaceuticals, Inc.
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