EMA Accepts Application for Breakthrough Treatment of Genetic Form of ALS

Anna Osborne
5 December 2022
Clinical Trials
Collaborations, Mergers & Acquisitions
Gene Therapy
Regulatory & Standards
Biogen, has announced the European Medicines Agency (EMA), has accepted its Marketing Authorization Application (MAA) for the review of torfersen – its investigational drug to treat superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS).

As a leading global biotech and pioneer in neuroscience, Biogen has been dedicated over the last decade to advancing research in all forms of ALS to develop potential targeted treatments.

ALS is a progressive and ultimately fatal neurodegenerative disease that causes the loss of motor neurons in the brain and spinal cord, resulting in a loss of voluntary muscle movement control. 

In around 2% of the approximately 168,000 people living with ALS globally, a mutation in the SOD1 gene is responsible. There are currently no treatments for ALS that target genetic causation, with some patients with this form of ALS surviving less than a year from symptom onset.

“Through our clinical development program, we have seen that torfersen has the potential to slow the progression of this relentless and ultimately fatal disease. 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,” said Priya Singhal, Head of Global Safety and Regulatory Sciences and Interim Head of R&D at Biogen.

Trofersen is an antisense drug developed, and currently under evaluation, by Biogen, as a treatment for SOD1-ALS. This product is designed to bind to SOD1 mRNA, inducing its degradation by RNase-H to reduce SOD1 protein production.

The Company has licensed torfersen from Ionis Pharmaceuticals under a collaborative development and license agreement.

Biogen’s EMA MAA for trofersen includes results from its Phase III trial VALOR, this studies ongoing open-label extension (OLE) study, as well as a Phase I study in healthy participants, and a Phase I/II investigating accelerating dose levels.

Also included in the EMA’S review are the most up-to-date 12-month integrated results VALOR and OLE, which have been published in The New England Journal of Medicine.

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In addition to VALOR, another Phase III trial – ATLAS – is currently evaluating trofersen’s potential to delay clinical onset in pre-symptomatic individuals with an identified SOD1 mutation and biomarker evidence of disease activity.

Alongside trofersen’s MAA review by the EMA, it is also under a Priority Review by the FDA, with a Prescription Drug User Fee Act action date of April 25, 2023. An early access program is also run by Biogen for torfersen, available in 34 countries.

“Today’s announcement is an important milestone for the ALS community in Europe where there is a tremendous need for additional treatment options,” reflects Philip Van Damme, Professor of Neurology and Director of the Neuromuscular Reference Center at the University Hospital Leuven in Belgium.

Source: Biogen Press Release

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