Qalsody (tofersen)
Intrathecal Vial (Ml)
Last reviewed · Source: First Databank (FDB) drug data
Overview
Tofersen is an antisense oligonucleotide.
Tofersen is a 20 base residue (20-mer) 5-10-5 MOE gapmer mixed backbone antisense oligonucleotide; the drug binds to SOD1 mRNA, causing its degradation and reduction in SOD1 protein synthesis.
Approximately 2% of all amyotrophic lateral sclerosis (ALS) cases and 15% of familial ALS cases are associated with mutations in SOD1, an antioxidant enzyme that protects the cell from reactive oxide species toxicity. Neuronal degeneration in ALS is thought to be caused by toxic gain of function of the mutant SOD1 protein. SOD1 has also been shown to potentially activate the transcription of nuclear genes and act as an RNA-binding protein.
Mutated SOD1 proteins accumulate within motor neurons and glial cells, leading to protein aggregation and the formation of toxic protein clumps, causing dysfunction in the axonal transport system. The exact mechanism of how the intracellular aggregates are toxic to motor neurons is not known. Tofersen treatment has been shown to result in a 35% reduction in total CSF SOD1 proteins at 28 weeks.
Following intrathecal injection, tofersen is distributed from the CSF to CNS tissue. The maximum concentration occurs after the third loading dose and there is little to no accumulation. The median time to peak plasma concentration is 2-6 hours.
The half life of tofersen is 4 weeks. The effects of sex, race, age, and body weight on tofersen exposure are not clinically significant. No clinical studies have been conducted to evaluate the pharmacokinetics of tofersen in patients with renal or hepatic impairment.
Tofersen is not expected to undergo metabolism by hepatic enzymes.
USES: Tofersen is used to treat a certain type of nerve disease called amyotrophic lateral sclerosis (ALS, also commonly called Lou Gehrig's disease).
Generic name: tofersen
Pronunciation: (toe-FER-sen)
Therapeutic class: Locomotor System
Reference product & biosimilars
No reference, biosimilar or interchangeable associated with this drug.
Today’s intelligence on Qalsody
Indications
The following indications are listed for Qalsody:
- Amyotrophic Lateral Sclerosis With Superoxide Dismutase Gene Mutation
Also known as: Als With Sod1 Gene Mutation.
Dosing
No dosing information is available for this tab.
Tofersen is administered intrathecally using a lumbar puncture.
| Drug label | Dosing type | Instructions |
|---|---|---|
| Qalsody 100 Mg/15 Ml Vial | Maintenance | Inject 15 milliliters (100 mg) by intrathecal route every 28 days. |
No dosing information is available for this tab.
Drug interactions
No clinical drug interaction studies have been performed with tofersen. Tofersen is not a substrate or inhibitor/inducer of major CYP enzymes, or a substrate or inhibitor of major transporters.
There are no contraindication interactions for this drug.
There are no severe interactions for this drug.
There are no moderate interactions for this drug.
Contraindications
*None.
There are no contraindication contraindications for this drug.
There are no severe contraindications for this drug.
- Papilledema
Adverse reactions
The most common adverse reactions (>=10% of patients treated with tofersen and greater than placebo) reported with tofersen in clinical studies were pain, fatigue, arthralgia, increased CSF white blood cells, and myalgia.
More Frequent
- Pleocytosis of cerebrospinal fluid
Less Frequent
- Elevated protein level in cerebrospinal fluid
- Neuralgia
Rare or Very Rare
- Intracranial hypertension
- Myelitis
- Non-infective meningitis
- Papilledema
- Radiculitis
More Frequent
- Arthralgia
- Fatigue
- Myalgia
- Pain
Less Frequent
- Muscle rigidity
Rare or Very Rare
- Fever
Precautions
No enhanced messaging available.
Safety and effectiveness of tofersen in pediatric patients have not been established.
There are no adequate data on developmental risks associated with the use of tofersen in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. Subcutaneous administration of tofersen in rabbits and mice resulted in no adverse effects on embryofetal development and pre- or postnatal development.
It is not known whether tofersen is distributed into human milk; the drug is distributed into milk in mice following subcutaneous administration. The effects of tofersen on nursing infants or on milk production are not known. Consider the benefits of breast-feeding along with the importance of tofersen to the woman and any potential adverse effects on the breast-fed infant from the drug or the underlying maternal condition.
Dosage adjustments are not needed in geriatric patients; however, greater sensitivity to the drug for some older individuals cannot be ruled out.
No overall differences in efficacy or safety of tofersen have been observed in geriatric patients compared with younger adults; however, the possibility that some geriatric patients may exhibit increased sensitivity to the drug cannot be ruled out.
ICD codes
| Code | Type | Description |
|---|---|---|
| Amyotrophic lateral sclerosis with superoxide dismutase gene mutation | ||
| G12.21 | ICD10CM | Amyotrophic lateral sclerosis |
ICD code information from First Databank (FDB).
Formulary status
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Last reviewed · Source: First Databank (FDB) drug data
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