Tofersen
Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Rithish Nimmagadda,MBBS.[2]
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Overview
Tofersen is an antisense oligonucleotide that is FDA approved for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene.. Common adverse reactions include he most common adverse reactions (≥ 10% of patients treated with tofersen and greater than placebo) were pain, fatigue, arthralgia, increased CSF white blood cell, and myalgia..
Adult Indications and Dosage
FDA-Labeled Indications and Dosage (Adult)
-Tofersen is administered intrathecally using a lumbar puncture by, or under the direction of, healthcare professionals experienced in performing lumbar punctures.
-Recommended dose is 100 mg (15 mL) per administration
-Initiate tofersen treatment with 3 loading doses administered at 14-day intervals. [ref] A maintenance dose should be administered once every 28 days thereafter.
-Allow vial to warm to room temperature prior to administration.
-Withdraw the required dose of 15 mL (equivalent to 100 mg) from the vial; do not dilute.
-Administer within 4 hours of removal from vial.
-Prior to administration, remove approximately 10 mL of CSF.
-Administer as an intrathecal bolus injection over 1 to 3 minutes.
-Consult prescribing information for additional instructions on preparation and administration of tofersen
Off-Label Use and Dosage (Adult)
Pediatric Indications and Dosage
FDA-Labeled Indications and Dosage (Pediatric)
There is limited information regarding Tofersen FDA-Labeled Indications and Dosage (Pediatric) in the drug label.
Off-Label Use and Dosage (Pediatric)
Contraindications
None
Warnings
-Myelitis and Radiculitis
Serious adverse reactions of myelitis and radiculitis have been reported in patients treated with tofersen. Six patients treated with tofersen experienced myelitis or radiculitis in the clinical studies. Two patients discontinued treatment with tofersen and required symptomatic management with full resolution of symptoms. In the remaining 4 patients, symptoms resolved without discontinuation of the drug. If symptoms consistent with myelitis or radiculitis develop, diagnostic workup and treatment should be initiated according to the standard of care. Management may require interruption or discontinuation of tofersen.
-Papilledema and Elevated Intracranial Pressure
Serious adverse reactions of papilledema and elevated intracranial pressure have been reported in patients treated with tofersen. Four patients developed elevated intracranial pressure and/or papilledema. All patients received treatment with standard of care with resolution of symptoms, and no events led to discontinuation of tofersen. If symptoms consistent with papilledema or elevated intracranial pressure develop, diagnostic workup and treatment should be initiated according to the standard of care.
-Aseptic Meningitis
Serious adverse reactions of aseptic meningitis (also called chemical meningitis or drug-induced aseptic meningitis) have been reported in patients treated with tofersen. One patient experienced a serious adverse reaction of chemical meningitis, which led to discontinuation of tofersen. One patient experienced a serious adverse reaction of aseptic meningitis, which did not lead to discontinuation of the drug. In addition, nonserious increases in CSF white blood cell and CSF protein have also been reported with tofersen. If symptoms consistent with aseptic meningitis develop, diagnostic workup and treatment should be initiated according to the standard of care.
Adverse Reactions
Clinical Trials Experience
There is limited information regarding Tofersen Clinical Trials Experience in the drug label.
Postmarketing Experience
There is limited information regarding Tofersen Postmarketing Experience in the drug label.
Drug Interactions
There is limited information regarding Tofersen Drug Interactions in the drug label.
Use in Specific Populations
Pregnancy
Pregnancy Category (FDA):
There is no FDA guidance on usage of Tofersen in women who are pregnant.
Pregnancy Category (AUS):
There is no Australian Drug Evaluation Committee (ADEC) guidance on usage of Tofersen in women who are pregnant.
Labor and Delivery
There is no FDA guidance on use of Tofersen during labor and delivery.
Nursing Mothers
There is no FDA guidance on the use of Tofersen in women who are nursing.
Pediatric Use
There is no FDA guidance on the use of Tofersen in pediatric settings.
Geriatic Use
There is no FDA guidance on the use of Tofersen in geriatric settings.
Gender
There is no FDA guidance on the use of Tofersen with respect to specific gender populations.
Race
There is no FDA guidance on the use of Tofersen with respect to specific racial populations.
Renal Impairment
There is no FDA guidance on the use of Tofersen in patients with renal impairment.
Hepatic Impairment
There is no FDA guidance on the use of Tofersen in patients with hepatic impairment.
Females of Reproductive Potential and Males
There is no FDA guidance on the use of Tofersen in women of reproductive potentials and males.
Immunocompromised Patients
There is no FDA guidance one the use of Tofersen in patients who are immunocompromised.
Administration and Monitoring
Administration
Inform patients and caregivers that tofersen 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 tofersen 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 tofersen could cause aseptic meningitis. Instruct patients and caregivers to contact their healthcare provider if symptoms consistent with meningitis develop.
Monitoring
There is limited information regarding Tofersen Monitoring in the drug label.
IV Compatibility
There is limited information regarding the compatibility of Tofersen and IV administrations.
Overdosage
There is limited information regarding Tofersen overdosage. If you suspect drug poisoning or overdose, please contact the National Poison Help hotline (1-800-222-1222) immediately.
Pharmacology
There is limited information regarding Tofersen Pharmacology in the drug label.
Mechanism of Action
There is limited information regarding Tofersen Mechanism of Action in the drug label.
Structure
There is limited information regarding Tofersen Structure in the drug label.
Pharmacodynamics
There is limited information regarding Tofersen Pharmacodynamics in the drug label.
Pharmacokinetics
There is limited information regarding Tofersen Pharmacokinetics in the drug label.
Nonclinical Toxicology
There is limited information regarding Tofersen Nonclinical Toxicology in the drug label.
Clinical Studies
There is limited information regarding Tofersen Clinical Studies in the drug label.
How Supplied
There is limited information regarding Tofersen How Supplied in the drug label.
Storage
There is limited information regarding Tofersen Storage in the drug label.
Images
Drug Images
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Package and Label Display Panel
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Patient Counseling Information
There is limited information regarding Tofersen Patient Counseling Information in the drug label.
Precautions with Alcohol
Alcohol-Tofersen interaction has not been established. Talk to your doctor regarding the effects of taking alcohol with this medication.
Brand Names
Qalsody®
Look-Alike Drug Names
There is limited information regarding Tofersen Look-Alike Drug Names in the drug label.
Drug Shortage Status
Price
References
The contents of this FDA label are provided by the National Library of Medicine.