Qalsody

Generic name: tofersen

Dosage form: intrathecal injection (100 mg/15 mL)

Drug class: Miscellaneous central nervous system agents

Dosage forms: Intrathecal injection.

Route of administration: Intrathecal (into the spinal canal).

Dose: Loading dose: 100 mg administered intrathecally once every 14 days for 3 doses. Maintenance dose: 100 mg administered intrathecally once every 28 days.

Mechanism of action: Tofersen is designed to reduce the production of superoxide dismutase 1 (SOD1) protein. Mutations in the SOD1 gene are linked to amyotrophic lateral sclerosis (ALS). By binding to SOD1 mRNA, tofersen prevents the production of the mutant SOD1 protein, potentially slowing the progression of ALS symptoms associated with SOD1 mutations.

Drug usage cases: Treatment of amyotrophic lateral sclerosis (ALS) associated with mutations in the SOD1 gene.

Drug contraindications: Known hypersensitivity to tofersen or any component of the formulation.

Side effects: Common: Headache, procedural pain, back pain, nausea, vomiting. Serious: Aseptic meningitis, increased cerebrospinal fluid protein levels, myelitis. Rare: Severe allergic reactions, increased liver enzymes, neurotoxicity.

Warnings: Monitor for signs of aseptic meningitis, such as headache, fever, stiff neck, nausea, vomiting, sensitivity to light, or altered mental status. Use with caution in patients with a history of meningitis or other infections of the central nervous system. Inform your doctor if you experience symptoms of an allergic reaction, such as rash, itching, swelling, or difficulty breathing. Regular monitoring of liver function and cerebrospinal fluid protein levels is recommended during treatment.

Use during pregnancy or breastfeeding: There is limited information on the use of tofersen during pregnancy. Use during pregnancy only if the potential benefit justifies the potential risk to the fetus. It is unknown if tofersen is excreted in human milk. Consult your doctor before using this medication while breastfeeding.