Tofersen

What is Tofersen?

• Tofersen (Qalsody), is an antisense oligonucleotide used for the treatment of amyotrophic lateral sclerosis (ALS).

What are the uses of this medicine?

• Tofersen (Qalsody) is used for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene.

How does this medicine work?

• Tofersen is an antisense oligonucleotide that causes degradation of SOD1 mRNA through binding to SOD1 mRNA, which results in a reduction of SOD1 protein synthesis.

Who Should Not Use this medicine ?

• This medicine have no usage limitations.

What drug interactions can this medicine cause?

• Tell your doctor and pharmacist what other prescription and nonprescription medications, vitamins, nutritional supplements, and grapefruit you are taking or plan to take.

Is this medicine FDA approved?

• Tofersen was approved for medical use in the United States in April 2023.

How should this medicine be used?

Recommended dosage:

• The recommended dosage is 100 mg (15 mL) of Qalsody per administration.
• Initiate Qalsody treatment with 3 loading doses administered at 14 day intervals. A maintenance dose should be administered once every 28 days thereafter.
• If the second loading dose is missed, administer Qalsody 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 Qalsody as soon as possible, and administer the next dose 28 days later.

Administration:

• Qalsody is supplied as a sterile, preservative-free, clear, and colorless to slightly yellow solution in a Type I glass vial to be administered by intrathecal administration.
• Administer Qalsody intrathecally using a lumbar puncture by, or under the direction of, healthcare professionals experienced in performing lumbar punctures.
• Allow to warm to room temperature prior to administration.
• Administer within 4 hours of removal from vial.
• Prior to administration, remove approximately 10 mL of cerebrospinal fluid.
• Administer as an intrathecal bolus injection over 1 to 3 minutes.

What are the dosage forms and brand names of this medicine?

This medicine is available in fallowing doasage form:

• As Injection: 100 mg/15 mL (6.7 mg/mL) solution in a single-dose vial.

This medicine is available in fallowing brand namesː

• Qalsody

What side effects can this medication cause?

The most common side effects of this medicine may include:

• Pain
• Fatigue
• Arthralgia
• CSF white blood cell increased
• Myalgia
• CSF protein increased
• Musculoskeletal stiffness
• Neuralgia

Less common, but serious side effects may include:

• Myelitis and/or Radiculitis
• Papilledema and Elevated Intracranial Pressure
• Aseptic Meningitis

What special precautions should I follow?

• Inform patients and caregivers that Qalsody could cause myelitis and radiculitis. Instruct patients and caregivers to contact their healthcare provider if symptoms consistent with these adverse reactions develop.
• Inform patients and caregivers that Qalsody could cause papilledema and elevated intracranial pressure. Instruct patients and caregivers to contact their healthcare provider if symptoms consistent with these adverse reactions develop.
• Inform patients and caregivers that Qalsody could cause aseptic meningitis. Instruct patients and caregivers to contact their healthcare provider if symptoms consistent with meningitis develop.
• There are no data on the presence of tofersen or its metabolites in human milk, the effects on the breastfed infant, or the effects on milk production.

What to do in case of emergency/overdose?

• In case of overdose, call the poison control helpline of your country. In the United States, call 1-800-222-1222.

• Overdose related information is also available online at poisonhelp.org/help.
• In the event that the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services. In the United States, call 911.

Can this medicine be used in pregnancy?

• There are no adequate data on developmental risks associated with the use of Qalsody in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes.

Can this medicine be used in children?

• Safety and effectiveness in pediatric patients have not been established.

What are the active and inactive ingredients in this medicine?

Active ingredient:

• tofersen

Inactive ingredients:

• sodium phosphate, monobasic, unspecified form
• sodium phosphate, dibasic, unspecified form
• sodium chloride
• potassium chloride
• calcium chloride
• magnesium chloride
• water

Who manufactures and distributes this medicine?

Manufactured by:

• Biogen MA Inc.
• Cambridge, MA

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

• Store refrigerated between 2°C to 8°C (36°F to 46°F) in the original carton to protect from light.
• Do not freeze.
• If no refrigeration is available, Qalsody may be stored in its original carton, protected from light at or below 30°C (86°F) for up to 14 days.
• If removed from the original carton, unopened vials of Qalsody can be removed from and returned to the refrigerator, if necessary, for not more than 6 hours per day at or below 30°C (86°F) for a maximum of 6 days (36 hours).

• Dailymed label info on Tofersen
• FDA Tofersen

Tofersen Resources
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