Qalsody

— THERAPEUTIC CATEGORIES —
  • Miscellaneous neurodegenerative disorders
PAGE CONTENTS
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  • Generic Name & Formulations
  • Indications
  • Dosage and Administration
  • Contraindications
  • Boxed Warnings
  • Warnings/Precautions
  • Pharmacokinetics
  • Interactions
  • Adverse Reactions
  • Clinical Trials
  • Note
  • Patient Counseling

    • Qalsody Generic Name & Formulations

    Legal Class

    Rx

    General Description

    Tofersen 6.7mg/mL; soln for intrathecal inj; preservative-free.

    Pharmacological Class

    Antisense oligonucleotide.

    How Supplied

    Single-dose vial (15mL)—1

    Storage

    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). 

    Manufacturer

    Biogen

    Generic Availability

    NO

    Mechanism of Action

    Tofersen causes degradation of SOD1 mRNA through binding to SOD1 mRNA, which results in a reduction of SOD1 protein synthesis.

    Qalsody Indications

    Indications

    Amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene.

    Qalsody Dosage and Administration

    Adult

    Remove ~10mL of CSF (via lumbar puncture needle) prior to administration. Give as an intrathecal bolus inj over 1–3mins (via lumbar puncture needle). Each dose is 100mg (15mL). Initiate with 3 loading doses given at 14-day intervals. Maintenance dose: give every 28 days thereafter.

    Children

    Not established.

    Qalsody Contraindications

    Not Applicable

    Qalsody Boxed Warnings

    Not Applicable

    Qalsody Warnings/Precautions

    Warnings/Precautions

    Monitor for myelitis and/or radiculitis, papilledema, elevated intracranial pressure, aseptic meningitis; evaluate and treat accordingly if symptoms occur. Pregnancy. Nursing mothers.

    Qalsody Pharmacokinetics

    Absorption

    Median time to maximum concentration (Tmax) plasma: range 2–6 hours.

    Metabolism

    Exonuclease (3'- and 5')-mediated hydrolysis.

    Elimination

    Half-life: ~4 weeks.

    Qalsody Interactions

    Not Applicable

    Qalsody Adverse Reactions

    Adverse Reactions

    Pain, fatigue, arthralgia, increased CSF white blood cell, myalgia, increased CSF protein, musculoskeletal stiffness, neuralgia.

    Qalsody Clinical Trials

    Clinical Trials

    The approval was based on data from the 28-week phase 3 VALOR study (ClinicalTrials.gov Identifier: NCT02623699), which compared the efficacy and safety of tofersen to placebo in 108 adults with ALS and a documented SOD1 mutation. Of these patients, 95 enrolled in the ongoing open-label extension (OLE) study (ClinicalTrials.gov Identifier: NCT03070119).

    The primary endpoint of the study was the change from baseline to week 28 in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R). While the results were not statistically significant, patients treated with tofersen experienced less decline from baseline in the ALSFRS-S compared with placebo (adjusted mean difference, 1.2 [95% CI, -3.2, 5.5]). Treatment with tofersen was associated with a nominally statistically significant reduction in total CSF SOD1 protein levels (P <.0001) and plasma NfL concentration (P <.0001) compared with placebo.

    Qalsody Note

    Not Applicable

    Qalsody Patient Counseling

    Cost Savings Program

    Qalsody Support and Resources

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