Biogen Announces 3-Month Extension of FDA Review Period

  • The new Prescription Drug User Fee Act (PDUFA) action date set by the FDA is April 25, 2023

CAMBRIDGE, Mass., Oct. 17, 2022 (GLOBE NEWSWIRE) — Biogen Inc. (Nasdaq: BIIB) today announced that the United States Food and Drug Administration (FDA) has extended the review period for three-month new drug application (NDA) for tofersen. Tofersen is an experimental superoxide dismutase 1 (SOD1) treatment for amyotrophic lateral sclerosis (ALS). The updated Prescription Drug User Fee Act (PDUFA) target date is April 25, 2023.

As part of the ongoing review, Biogen has submitted responses to FDA information requests that the FDA has considered a major amendment to the application that will require additional time for review.

“We are committed to providing all the details the agency needs to complete tofersen’s review,” said Priya Singhal, MD, MPH, head of global safety and regulatory science and acting head of R&D at Biogen. “As the review continues, Biogen will maintain the early access program for tofersen.”

The FDA accepted the tofersen NDA in July 2022 under the fast track and granted priority review.

About Tofersen
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 Pharmaceuticals, Inc. 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.1

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 around 2% of the approximately 168,000 people with ALS worldwide (SOD1-ALS).2 Life expectancy in SOD1-ALS varies widely, with some patients surviving less than a year.3

Biogen’s ongoing commitment to ALS
For more than a decade, Biogen has been committed to advancing ALS research to better understand all forms of the disease. The company continued to invest in pioneering research despite the difficult decision to discontinue an advanced-stage ALS asset in 2013. Biogen applied significant learnings to its portfolio of assets for genetic ALS and other forms, in the goal of increasing the likelihood of bringing potential therapy to patients who need it. These applied learnings include the evaluation of genetically validated targets in defined patient populations, the pursuit of the most appropriate modality for each target, and the use of sensitive clinical parameters. Today, the company has a pipeline of investigational drugs being evaluated in ALS, including tofersen and BIIB105.

About Biogene
As pioneers in neuroscience, Biogen discovers, develops and delivers innovative therapies worldwide for people living with serious neurological diseases and related therapeutic adjacencies. One of the world’s first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Sir Kenneth Murray and Nobel laureates Walter Gilbert and Phillip Sharp. Today, Biogen has a leading portfolio of drugs to treat multiple sclerosis, introduced the first approved therapy for spinal muscular atrophy, and developed the first and only approved therapy to treat a defining disease-defining pathology. Alzheimer’s. Biogen also commercializes biosimilars and is focused on advancing one of the industry’s most diverse neuroscience pipelines that will transform the standard of care for patients in several areas of high unmet need.

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 in all company operations, establish collaborations with renowned institutions to advance science to improve human health outcomes and support underserved communities. served.

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Biogen Safe Harbor
This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including statements about the results of the Phase 3 VALOR study of tofersen or its OLE; the potential clinical effects of tofersen; the potential benefits, safety and efficacy of tofersen; the clinical development program for tofersen; tofersen’s potential endorsement; identification and treatment of ALS; our research and development program for the treatment of ALS; the potential of our business operations and pipeline programs, including tofersen; and the risks and uncertainties associated with drug development and commercialization. These forward-looking statements may be accompanied by words such as “aims”, “anticipates”, “believes”, “could”, “estimates”, “expects”, “plans”, “intends”, ” can”, “plan”. , “”potential”, “possible”, “will”, “would” and other words and terms of similar meaning. The development and commercialization of drugs involves a high degree of risk and only a small number of research programs and development lead to commercialization of a product. Results of early-stage clinical trials may not be indicative of full results or results of later-stage or larger-scale clinical trials and do not guarantee the Regulatory Approval You should not place undue reliance on these statements or the scientific data presented.

These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in these statements, including, without limitation, the uncertainty of success in the development and potential commercialization of tofersen; the risk that we may not be able to fully enroll our clinical trials or that enrollment may 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 delay approval of our drug candidates, including tofersen; the occurrence of adverse safety events; risks of unforeseen obstacles, costs or delays; failure to protect and enforce our data, intellectual property and other proprietary rights and uncertainties related to intellectual property claims and disputes; 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, 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 United States Securities and Exchange Commission. These statements are based on our current beliefs and expectations and speak only as of the date of this press release.

We undertake no obligation to publicly update any forward-looking statements, whether as a result of new information, future developments or otherwise.


  1. Brown RH, Al-Chalabi A. Amyotrophic lateral sclerosis. N Engl J Med. July 13, 2017
  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
  3. Bali T, et al. Defining the natural history of SOD1 ALS to guide the design of therapeutic clinical trials. J Neurol Neurosurg Psychiatry. 2017 February

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