Tofersen at a glance
Medicine class
Antisense oligonucleotide genetic therapy
Licensed use
SOD1-associated amyotrophic lateral sclerosis in adults
Administration
Injection into spinal fluid through a lumbar puncture
Treatment setting
Given by healthcare professionals experienced in lumbar puncture
What is Tofersen used for?
The UK-licensed indication is:
Treatment of adults with amyotrophic lateral sclerosis associated with an SOD1 gene mutation.
How does Tofersen work?
Tofersen binds to the messenger RNA carrying instructions for SOD1 protein. The messenger RNA is then broken down, reducing production of the abnormal SOD1 protein associated with motor-neurone damage.
Tofersen preparations and strengths
Injection
Route: Parenteral
Strengths: 100 mg
How to take Tofersen
Tofersen is not swallowed or self-injected. A specialist healthcare professional injects it into the fluid surrounding the spinal cord through a lumbar puncture. Cerebrospinal fluid is removed beforehand; image guidance or sedation may be considered when appropriate.
Attend the specialist unit as arranged.
Tell the team about new symptoms, medicines and previous difficulties with lumbar puncture.
Follow the unit's instructions after the procedure.
Tofersen side effects
Common or expected effects
- Pain, including back or limb pain
- Joint or muscle pain
- Tiredness
- Fever
- Muscle stiffness
- Nerve pain, burning or pins and needles
- Headache or back pain associated with lumbar puncture
- Increased protein or white blood cells in cerebrospinal fluid
Get urgent medical advice
- New or worsening weakness, numbness, abnormal sensations or pain
- Blurred or double vision, loss of vision or severe headache
- Headache with fever, neck stiffness, nausea or vomiting
- Symptoms suggesting infection after lumbar puncture
Tofersen in pregnancy
Human pregnancy data are unavailable. Tofersen is not recommended during pregnancy or for someone who could become pregnant and is not using contraception. Seek specialist advice when pregnancy is planned or suspected.
Tofersen while breastfeeding
It is unknown whether tofersen passes into human milk, and risk to a breastfed child cannot be excluded. The specialist and patient should decide whether to stop breastfeeding or avoid tofersen after weighing the benefits of each.
Tofersen interactions
Formal interaction studies have not been performed. Tell the specialist team about all medicines being used.
Other intrathecal medicines
Combined use has not been evaluated, so its safety is unknown and requires specialist review.
Common questions about Tofersen
Answers are fully visible for fast scanning and source review.
Who can receive tofersen in the UK?
It is licensed for adults with amyotrophic lateral sclerosis associated with an SOD1 gene mutation.
Does tofersen treat every type of motor neurone disease?
No. It specifically targets disease caused by an SOD1 mutation.
Is genetic testing needed before tofersen?
Yes. The licensed indication requires confirmation that ALS is associated with an SOD1 mutation.
Is tofersen a cure?
No. It lowers SOD1 protein production but does not reverse established disease, and response varies.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.