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Innovation Observatory > Reports > Drugs > Tofersen for the treatment of amyotrophic lateral sclerosis caused by mutations in the SOD1 gene

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Tofersen for the treatment of amyotrophic lateral sclerosis caused by mutations in the SOD1 gene

Drugs

Neurology and Neurosurgery

February 2020


Tofersen (BIIB067) is in clinical development for the treatment of amyotrophic lateral sclerosis (ALS – also known as motor neurone disease) caused by mutations in the SOD1 gene (SOD1-ALS). ALS is a progressive disease of the nervous system, where nerve cells in the brain and spinal cord that control voluntary movement gradually deteriorate, causing loss of muscle function and paralysis. ALS is a debilitating and life-threatening disease. The gradual loss of neurons leads to a paralysing effect on muscles used for breathing, which usually leads to death from respiratory failure.
Tofersen is designed to help some patients with ALS who have a change in the gene responsible for producing the enzyme SOD1. Mutations in the SOD1 gene confer a new toxic property to the SOD1 protein, though exactly how disease-associated SOD1 is toxic to neurons is unknown. Tofersen is made of a small strand of synthetic genetic material that prevents translation of SOD1. By reducing the amount of both normal and defective SOD1, tofersen is expected to improve the progression of SOD1-ALS. Tofersen is given as an intrathecal injection and if licensed, it will offer a treatment option for patients with SOD1-ALS who currently have very limited available options.

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