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tofersen (Qalsody)
Indications:
- treatment of amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene
Dosage:
- intrathecal administration
- 100 mg (15 mL) per administration
- initiate with 3 loading doses administered at 14 day intervals
- maintenance dose every 28 days
Injection: 6.7 mg/mL (15 mL)
Adverse effects:
- > 10%
- fatigue, arthralgia, myalgia, increased CSF leukocyte count
* serious events of myelitis & radiculitis
* papilledema & increased intracranial pressure
* aseptic meningitis
Mechanism of action:
- antisense oligonucleotide that targets mutated SOD1 genes to prevent expression of toxic SOD1 proteins
General
neurologic agent
Database Correlations
PUBCHEM cid=5282398
References
- ALS Association
BIOGEN IN PARTNERSHIP WITH IONIS PHARMACEUTICALS
Tofersen
https://www.als.org/navigating-als/living-with-als/fda-approved-drugs/tofersen
- HIGHLIGHTS OF PRESCRIBING INFORMATION
QALSODY (tofersen) injection, for intrathecal use.
https://www.biogencdn.com/us/pdfs/qalsody-prescribing-information.pdf