European Medicines Agency Accepts Tofersen Marketing Authorization Application to Treat Rare, Genetic Form of ALS
CAMBRIDGE, Mass., Dec. 05, 2022 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) announced the European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) 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 less than 1,000 people in Europe.2 There is currently no treatment targeted for SOD1-ALS.3
“Through our clinical development program, we have seen that tofersen has the potential to slow the progression of this relentless and ultimately fatal disease,” said Priya Singhal, M.D., M.P.H., Head of Global Safety and Regulatory Sciences and Interim Head of R&D at Biogen. “Regulatory submissions in the U.S. and now EU represent an important step in our efforts to bring the first genetically-targeted treatment for SOD1-ALS to the ALS community as quickly as possible.”
The MAA includes results from the Phase 3 VALOR study, its open label extension (OLE) study, a Phase 1 study in healthy volunteers and a Phase 1/2 study evaluating ascending dose levels. Also included are the most current 12-month integrated results from VALOR and the OLE study that were recently published in The New England Journal of Medicine.
“The effects we have seen with tofersen diverge from the natural history of the disease and bring hope for the treatment of SOD1-ALS,” said Philip Van Damme, M.D., Ph.D., professor of neurology and director of the Neuromuscular Reference Center at the University Hospital Leuven in Belgium. “Today’s announcement is an important milestone for the ALS community in Europe where there is a tremendous need for additional treatment options.”
Tofersen is also under review with the U.S. Food and Drug Administration with Priority Review and has a Prescription Drug User Fee Act action date of April 25, 2023.
Biogen will maintain its early access program for tofersen, which is now available in 34 countries. Biogen continues to actively engage with other regulators around the world and will provide updates when appropriate.
About Amyotrophic Lateral Sclerosis and SOD1-ALS
Multiple genes have been implicated in ALS. Genetic testing helps determine if a person’s ALS is associated with a genetic mutation, even in individuals without a 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 percent of the estimated 168,000 people who have ALS globally (SOD1-ALS).2 Life expectancy in SOD1-ALS varies widely with some patients surviving less than a year.4
Biogen’s Continuous Commitment to ALS
In 2020, Biogen launched a bold 20-year, $250 million initiative to address the deeply interrelated issues of climate, health, and equity. Healthy Climate, Healthy Lives™ aims to eliminate fossil fuels across the company’s operations, build collaborations with renowned institutions to advance the science to improve human health outcomes, and support underserved communities.
Biogen Safe Harbor
These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including without limitation, uncertainty of success in the development and potential commercialization of tofersen; the risk that we may not fully enroll our clinical trials or enrollment will take longer than expected; unexpected concerns may arise from additional data, analysis or results obtained during our clinical trials; regulatory authorities may require additional information or further studies, or may fail or refuse to approve or may delay approval of our drug candidates, including tofersen; the occurrence of adverse safety events; the risks of unexpected hurdles, costs or delays; failure to protect and enforce our data, intellectual property and other proprietary rights and uncertainties relating to intellectual property claims and challenges; product liability claims; and the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations and financial condition. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from our expectations in any forward-looking statement. Investors should consider this cautionary statement, as well as the risk factors identified in our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These statements are based on our current beliefs and expectations and speak only as of the date of this news release.
We do not undertake any obligation to publicly update any forward-looking statements, whether as a result of new information, future developments or otherwise.
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