Tofersen

Tofersen is used in adults to treat specific types of amyotrophic lateral sclerosis (ALS).

Your doctor will ensure you have the right type to be treated with tofersen.

Tofersen may also be used for reasons that are not mentioned in this medication guide.

Your doctor will do tests to confirm that tofersen is the right treatment for you.

Tofersen is injected into the fluid around the spinal cord. A healthcare provider will give you this injection.

Tofersen is given first as 3 injections every 14 days, then as maintenance injections every 28 days.

You may receive a sedative to help you relax during the injection.

After getting an injection, you will be monitored closely to ensure you do not have serious side effects.

You will need frequent medical tests.

Tell your doctor if you use other medicines or have other medical conditions or allergies.

Tell your doctor about all your medical conditions or allergies before receiving tofersen.

It is not known if tofersen will harm an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant.

Ask a doctor if it is safe to breastfeed while receiving tofersen.

Get emergency medical help if you have signs of an allergic reaction: hives, trouble breathing, swelling of your face, lips, tongue, or throat.

Some side effects may happen during the injection. Tell your medical caregiver if you feel dizzy, nauseous, light-headed, itchy, sweaty, or have a headache, chest tightness, back pain, trouble breathing, or swelling in your face.

Tofersen may cause serious side effects. Call your doctor right away if you have:

• severe muscle pain, cramps;
• signs of nerve pain--numbness, weakness, tingling, burning pain;
• signs of meningitis--fever, headache, neck stiffness, increased sensitivity to light, nausea, vomiting, confusion, drowsiness;
• signs of an eye condition called papilledema--blurry vision, swelling, vision changes; or
• increased pressure inside the skull--severe headaches, ringing in your ears, dizziness, nausea, vision problems, pain behind your eyes.

Your treatments may be delayed or permanently stopped if you have certain side effects.

Common side effects of tofersen may include:

• muscle or joint pain, muscle stiffness;
• feeling very tired;
• nerve or back pain;
• pain in your hands or feet; or
• increased white blood cells and protein in fluid around the spine.

This is not a complete list of side effects and others may happen. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Usual Adult Dose for Amyotrophic Lateral Sclerosis:

Loading dose: 100 mg intrathecally every 14 days for 3 dosesMaintenance dose: 100 mg intrathecally every 28 days Comments:-Consider utilizing patient sedation and/or imaging techniques to facilitate administration of this drug. -Prior to administration, remove 10 mL from cerebrospinal fluid and administer as an intrathecal bolus injection over 1 to 3 minutes using a lumbar puncture needle. -If the second loading dose is missed, administer it as soon as possible, and administer the third loading dose 14 days later.-If the third loading dose or a maintenance dose is missed, administer it as soon as possible, and administer the next dose 28 days later. Use: For the treatment of amyotrophic lateral sclerosis in adults who have a mutation in the superoxide dismutase 1 gene. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).

Call your doctor for instructions if you miss an appointment for your Tofersen injection.

In a medical setting, an overdose will be treated right away.

Follow your doctor's instructions regarding any limits on food, drinks, or activities.

Other drugs may affect tofersen, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all other medicines you use.