Why is this medication prescribed?

Tofersen Injection, also known by its generic name, tofersen, is a medication used in the treatment of a specific genetic disorder called amyotrophic lateral sclerosis (ALS). ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord, leading to muscle weakness, paralysis, and eventually respiratory failure. Tofersen works by targeting a specific genetic mutation associated with ALS, known as the C9orf72 mutation, and aims to slow down the progression of the disease.

How should this medicine be used?

Tofersen Injection, also known by its generic name, tofersen, is a medication prescribed for the treatment of amyotrophic lateral sclerosis (ALS) in patients with a specific genetic mutation known as the C9orf72 mutation. Here is how Tofersen Injection is typically used:

• Administration: Tofersen Injection is administered via intrathecal injection, which means it is injected directly into the spinal fluid through a lumbar puncture. This route of administration ensures that the medication reaches the central nervous system, where it can exert its therapeutic effects.
• Healthcare Professional Administration: Tofersen Injection should be administered by a healthcare professional who is experienced in performing lumbar punctures and intrathecal injections. This ensures proper administration technique and minimizes the risk of complications.
• Dosage: The dosage of Tofersen Injection is determined by the prescribing healthcare provider based on factors such as the patient’s age, weight, renal function, and the severity of ALS. The dosage may be adjusted over time based on the patient’s response to treatment and any adverse effects experienced.
• Frequency of Administration: The frequency of Tofersen Injection administration also depends on the prescribing healthcare provider’s recommendations. Typically, Tofersen Injection is administered at regular intervals as prescribed by the healthcare provider.
• Monitoring: Patients receiving Tofersen Injection should be closely monitored by their healthcare provider for any signs of improvement in ALS symptoms, as well as for any adverse effects or complications associated with the medication. Regular follow-up visits may be scheduled to assess treatment response and adjust the dosage or frequency of administration if necessary.
• Patient Education: Patients and caregivers should receive thorough instructions from their healthcare provider regarding the administration technique, proper handling of the medication, and any precautions or warning signs to watch out for. It’s essential to follow these instructions carefully to ensure the safe and effective use of Tofersen Injection.

It’s crucial for patients to adhere to their prescribed treatment regimen and attend all scheduled appointments with their healthcare provider for monitoring and follow-up care. Additionally, patients should report any new or worsening symptoms, as well as any adverse effects or concerns, to their healthcare provider promptly.

Other uses for this medicine

Tofersen Injection (tofersen) is primarily indicated for the treatment of amyotrophic lateral sclerosis (ALS) in patients with a specific genetic mutation known as the C9orf72 mutation. There are no other approved uses for Tofersen Injection beyond ALS with this specific genetic mutation.

What special precautions should I follow?

Regarding special precautions for Tofersen Injection, it’s important to follow these guidelines:

• Administration by Healthcare Professionals: Tofersen Injection should only be administered by healthcare professionals who are experienced in performing lumbar punctures and intrathecal injections. This ensures proper administration technique and reduces the risk of complications.
• Monitoring: Patients receiving Tofersen Injection should be closely monitored by their healthcare provider for any signs of improvement in ALS symptoms, as well as for any adverse effects or complications associated with the medication. Regular follow-up visits may be scheduled to assess treatment response and adjust the dosage or frequency of administration if necessary.
• Allergic Reactions: Patients should inform their healthcare provider of any known allergies or hypersensitivity reactions before receiving Tofersen Injection. Although rare, allergic reactions to medications can occur, and prompt medical attention should be sought if signs of an allergic reaction develop, such as rash, itching, swelling, dizziness, or difficulty breathing.
• Drug Interactions: Patients should inform their healthcare provider about all medications, supplements, and herbal products they are taking before starting Tofersen Injection. Certain medications may interact with Tofersen, potentially affecting its effectiveness or increasing the risk of adverse effects. Your healthcare provider can determine if any adjustments to your medication regimen are necessary.
• Pregnancy and Breastfeeding: The safety of Tofersen Injection during pregnancy and breastfeeding has not been established. Patients should discuss the risks and benefits of Tofersen with their healthcare provider if they are pregnant, planning to become pregnant, or breastfeeding.
• Driving and Operating Machinery: Tofersen Injection may cause dizziness or drowsiness in some patients. Patients should exercise caution when driving, operating machinery, or engaging in activities that require mental alertness until they know how Tofersen affects them.

Patients should always follow their healthcare provider’s instructions and report any new or worsening symptoms, as well as any concerns or questions about Tofersen Injection.

What special dietary instructions should I follow?

There are no specific dietary instructions associated with Tofersen Injection. However, maintaining a balanced and nutritious diet as recommended by your healthcare provider is essential for overall health and well-being, especially for individuals with ALS.

What should I do if I forget a dose?

If you miss a dose of Tofersen Injection, contact your healthcare provider for guidance. They will advise you on the appropriate course of action, which may include rescheduling the missed dose or adjusting your treatment plan accordingly. It’s essential to follow your healthcare provider’s instructions closely to ensure the effectiveness of your treatment. Avoid doubling up on doses to make up for a missed dose unless specifically instructed to do so by your healthcare provider.

What side effects can this medication cause?

Tofersen Injection, like any medication, can potentially cause side effects. Not all people will experience these side effects, and their severity can vary. Common side effects associated with Tofersen Injection may include:

• Headache
• Back pain
• Pain at the injection site
• Fall
• Injury
• Post-lumbar puncture syndrome (a group of symptoms, including headache, neck stiffness, nausea, vomiting, and dizziness, that can occur after a lumbar puncture)

It’s important to note that this list may not be exhaustive, and other side effects may occur. Additionally, some side effects may be serious and require medical attention. If you experience any unusual or severe symptoms after receiving Tofersen Injection, you should contact your healthcare provider immediately.

As with any medication, the benefits of Tofersen Injection in treating ALS with the C9orf72 mutation should be carefully weighed against the potential risks of side effects. Your healthcare provider can provide more information about the potential side effects of Tofersen Injection and help you make an informed decision about your treatment.

What should I know about storage and disposal of this medication?

Regarding the storage and disposal of Tofersen Injection:

Storage:

• Tofersen Injection should be stored in the refrigerator at a temperature between 2°C to 8°C (36°F to 46°F). Do not freeze.
• Keep Tofersen Injection in its original carton to protect it from light.
• Do not shake the vial. Gently swirl the vial to dissolve any particles before use.
• Do not use Tofersen Injection if it has been frozen or if it contains particles or is discolored.

Disposal:

• Unused or expired Tofersen Injection should be disposed of properly according to local regulations.
• Do not dispose of Tofersen Injection in household trash. Consult your healthcare provider or pharmacist for guidance on proper disposal methods.

In case of emergency/overdose

• If you suspect an overdose or experience severe adverse effects after receiving Tofersen Injection, seek emergency medical attention immediately.
• Contact your local poison control center or emergency room for further guidance.

What other information should I know?

• Keep all appointments with your healthcare provider for monitoring and follow-up care while receiving Tofersen Injection.
• Inform your healthcare provider about all medications, supplements, and herbal products you are taking, as well as any medical conditions you have, before starting Tofersen Injection.
• Follow your healthcare provider’s instructions carefully regarding the dosage and administration of Tofersen Injection.
• Report any new or worsening symptoms, as well as any concerns or questions about Tofersen Injection, to your healthcare provider promptly.

It’s essential to follow all instructions provided by your healthcare provider and pharmacist for the safe and effective use of Tofersen Injection. If you have any questions or concerns about Tofersen Injection, don’t hesitate to discuss them with your healthcare provider.