Version May 2024
Missed doses:
Second loading dose: Administer missed dose as soon as possible, and administer third loading dose 14 days later.
Third loading dose or maintenance dose: Administer missed dose as soon as possible, and administer next dose 28 days later.
There are no dosage adjustments provided in the manufacturer's labeling (has not been studied).
There are no dosage adjustments provided in the manufacturer's labeling (has not been studied); not expected to undergo metabolism by hepatic enzymes.
Myelitis and/or radiculitis, symptoms: Evaluate and treat as clinically appropriate; tofersen interruption or discontinuation may be necessary.
Refer to adult dosing.
Serious neurologic events including myelitis, aseptic meningitis, nerve root disorder (radiculitis), increased intracranial pressure, abnormal proteins in cerebrospinal fluid (pleocytosis), and papilledema have been reported with tofersen. In a clinical study, most events resolved without drug discontinuation; however, one participant developed myelitis within 1 week of tofersen initiation which required drug discontinuation and treatment with glucocorticoid and plasma exchange; within 3 months, symptoms and imaging findings had resolved (Ref).
Mechanism: Not clearly established; the relationship between pleocytosis and serious neurologic events has not been elucidated (Ref).
The following adverse drug reactions and incidences are derived from product labeling unless otherwise specified. Adverse reactions reported in adults.
>10%:
Hematologic & oncologic: Leukocytosis (cerebrospinal fluid: 14%)
Nervous system: Fatigue (17%), pain (42%)
Neuromuscular & skeletal: Arthralgia (14%), myalgia (14%)
1% to 10%:
Nervous system: Abnormal proteins in cerebrospinal fluid (increased: 8%) (table 1), neuralgia (6%)
|
Drug (Tofersen) |
Placebo |
Number of Patients (Tofersen) |
Number of Patients (Placebo) |
Comments |
|---|---|---|---|---|
|
8% |
3% |
72 |
36 |
Increased cerebrospinal fluid protein |
Neuromuscular & skeletal: Muscle rigidity (6%)
Frequency not defined:
Nervous system: Aseptic meningitis, increased intracranial pressure, nerve root disorder (radiculitis)
Neuromuscular & skeletal: Myelitis
Ophthalmic: Papilledema
There are no contraindications listed in the manufacturer's labeling.
Excipient information presented when available (limited, particularly for generics); consult specific product labeling.
Solution, Intrathecal [preservative free]:
Qalsody: 100 mg/15 mL (15 mL)
No
Solution (Qalsody Intrathecal)
100 mg/15 mL (per mL): $1,138.40
Disclaimer: A representative AWP (Average Wholesale Price) price or price range is provided as reference price only. A range is provided when more than one manufacturer's AWP price is available and uses the low and high price reported by the manufacturers to determine the range. The pricing data should be used for benchmarking purposes only, and as such should not be used alone to set or adjudicate any prices for reimbursement or purchasing functions or considered to be an exact price for a single product and/or manufacturer. Medi-Span expressly disclaims all warranties of any kind or nature, whether express or implied, and assumes no liability with respect to accuracy of price or price range data published in its solutions. In no event shall Medi-Span be liable for special, indirect, incidental, or consequential damages arising from use of price or price range data. Pricing data is updated monthly.
Availability limited to Optum Frontier Therapies Specialty Distributor (1-833-754-6457) via Optum Frontier Therapies Specialty Pharmacy (1-855-768-9727). Additional access information is available at https://www.qalsodyhcp.com.
Intrathecal: Allow refrigerated vial to reach room temperature prior to administration; do not use external heat sources. Do not shake. Prior to administration, remove ~10 mL cerebrospinal fluid using a lumbar puncture needle. Attach a needle to a syringe and withdraw 15 mL (100 mg) tofersen from vial by inserting the syringe needle through center of the vial overseal. Administer with a lumbar puncture needle as an intrathecal bolus injection over 1 to 3 minutes. Tofersen contains no preservatives. Once drawn into the syringe, administer immediately (within 4 hours of removal from the vial) at room temperature; otherwise, it must be discarded. Do not dilute tofersen; external filters are not required. Discard any unused contents from vial.
Amyotrophic lateral sclerosis: Treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. Note: Approved under accelerated approval based on reduction in plasma neurofilament light chain; continued approval may be contingent upon verification of clinical benefit in confirmatory trials.
None known.
There are no known significant interactions.
Adverse events were not observed following SUBQ administration of tofersen in animal reproduction studies.
It is not known if tofersen is present in breast milk.
According to the manufacturer, the decision to breastfeed during therapy should consider the risk of infant exposure, the benefits of breastfeeding to the infant, and the benefits of treatment to the mother.
Signs and symptoms of neurological events, including aseptic meningitis, elevated intracranial pressure, papilledema, myelitis, and radiculitis.
Anti-sense oligonucleotide that induces ribonuclease H-mediated degradation of superoxide dismutase 1 (SOD1) mRNA, resulting in SOD1 protein synthesis reduction (Miller 2022; manufacturer’s labeling).
Distribution: Within CNS tissue.
Metabolism: Via exonuclease (3'- and 5')-mediated hydrolysis.
Half-life elimination: ~4 weeks (cerebrospinal fluid).
Time to peak: 2 to 6 hours (plasma).
آیا می خواهید مدیلیب را به صفحه اصلی خود اضافه کنید؟
