Tofersen Application Scope

Tofersen, marketed under the brand name QALSODY, is an antisense oligonucleotide indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults possessing a mutation in the superoxide dismutase 1 (SOD1) gene.

This approval is granted under accelerated approval based on the observed reduction in plasma neurofilament light chain (NfL) levels in patients treated with QALSODY.

Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trials.

tofersen

Tofersen Characteristics:

Ingredients: Tofersen is a synthetic antisense oligonucleotide designed to bind to SOD1 mRNA, reducing the production of SOD1 protein.
Properties: As an antisense oligonucleotide, tofersen decreases the production of the SOD1 protein, mutations of which are associated with ALS.
Specification: Each vial contains 100 mg/15 mL (6.7 mg/mL) of tofersen.
Packaging Specification: Supplied as a single-dose vial containing 15 mL of solution.
Storage: Store refrigerated at 2°C to 8°C (36°F to 46°F) in the original carton to protect from light. Do not freeze.
Expiry Date: Refer to the expiration date printed on the packaging.
Executive Standard: Approved under accelerated approval based on reduction in plasma NfL levels; continued approval may depend on confirmatory trials.
Approval Number: NDA #215887
Date of Revision: April 2023
Manufacturer: Biogen Inc.

Guidelines for the Use of Tofersen

Dosage and Administration:

Recommended Dosage: Administer QALSODY intrathecally. The dosing regimen consists of three loading doses of 100 mg each, administered at 14-day intervals, followed by a maintenance dose of 100 mg every 28 days.
Administration: QALSODY is administered intrathecally by or under the direction of healthcare professionals experienced in performing lumbar punctures.

Adverse Reactions:

Common adverse reactions (≥10% of patients) include:

Fatigue
Arthralgia (joint pain)
Increased cerebrospinal fluid white blood cell count
Myalgia (muscle pain)

Serious adverse reactions may include myelitis, increased intracranial pressure, and aseptic meningitis.

Contraindications:

QALSODY is contraindicated in patients with known hypersensitivity to tofersen or any of its components.

Precautions:

Neurologic Monitoring: Monitor for signs and symptoms of myelitis and increased intracranial pressure.
Aseptic Meningitis: Patients should be observed for symptoms of aseptic meningitis, such as headache, fever, and stiff neck.

Tofersen Interactions

Drug Interactions:

No formal drug interaction studies have been conducted with QALSODY. Given its mechanism of action and route of administration, the potential for drug-drug interactions is considered low.

Note:

If there is a new packaging for the drug, the new packaging shall prevail. The above information is sourced from HongKong DengYue Medicine. It is only for internal discussion among medical staff and does not serve as a basis for medication. For specific medication guidelines, please consult the attending physician.