Qalsody (tofersen) is a prescription medicine used to treat amyotrophic lateral sclerosis (ALS) in adults who have a specific genetic mutation in the superoxide dismutase 1 (SOD1) gene. Qalsody is the first FDA-approved treatment that targets a genetic cause of ALS to slow progression of ALS. Qalsody works by reducing the amount of a toxic protein (SOD1 protein) that causes damage to motor neurons and the symptoms of ALS .

ALS  patients who have a mutation in the SOD1 gene (SOD1-ALS) make a toxic misfolded form of SOD1 protein that damages the motor neurons which causes muscle weakness, loss of function, and in time even death. Qalsody is an antisense oligonucleotide (ASO) that lowers the amount the toxic SOD1 protein made by binding to SOD1 mRNA, which may help slow the progression of ALS.

Qalsody can cause serious side effects, contact your doctor immediately if you have:

Inflammation of the spinal cord (myelitis) and/ or irritation of the nerve roots (radiculitis) can occur in patients treated with Qalsody. Discuss with your healthcare provider to learn about the symptoms associated with myelitis or radiculitis. Treatment with Qalsody may need to be interrupted or discontinued.

Swelling of the optic nerve (papilledema) and increased pressure inside the skull (elevated intracranial pressure ) can occur in patients treated with Qalsody.  Discuss with your healthcare provider what the symptoms associated with papilledema or elevated intracranial pressure are.

Inflammation of the brain linings (aseptic meningitis) can occur in patients treated with Qalsody in patients treated with Qalsody. Discuss with your healthcare provider to learn more about symptoms associated with aseptic meningitis.

 Qalsody is administered intrathecally using a lumbar puncture, this means that it is given as an injection into the spinal canal.

Loading dose: 100 mg intrathecally every 14 days for 3 doses

Maintenance dose: 100 mg intrathecally every 28 days

Comments:

• Consider utilizing patient sedation and/or imaging techniques to facilitate administration of this drug.
•  Before administering this medicine remove 10 mL from cerebrospinal fluid and then administer Qalsody as an intrathecal bolus injection over 1 to 3 minutes using a lumbar puncture needle.
• If the second loading dose is missed, administer it as soon as possible, and administer the third loading dose 14 days later.
• If the third loading dose or a maintenance dose is missed, administer it as soon as possible, and administer the next dose 28 days later.

Use: For the treatment of amyotrophic lateral sclerosis in adults who have a mutation in the superoxide dismutase 1 gene. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).

Dosage form: Injection 100 mg/15 mL (6.7 mg/mL) solution in a single-dose vial that is free of preservatives.

Before taking this medicine it is important to tell your healthcare provider if you are pregnant or planning to become pregnant or are breastfeeding or planning to breastfeed.

Common Qalsody side effects may include:

• pain including back pain and pain in arms or legs
• feeling tired
• muscle and joint pain
• increased white blood cell count in the cerebrospinal fluid (CSF).

Less Common but serious side effects include:

• myelitis and radiculitis (see warnings above)
• papilledema and elevated intracranial pressure (see warnings above)
• aseptic meningitis (see warnings above)