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SOD1-ALS and NfL

The role of genetics and neurofilament in SOD1-ALS

SOD1-ALS is caused by mutations in the SOD1 gene that lead to accumulation of SOD1 protein, eventually resulting in neurodegeneration, weakness, and death1,2

Studies show that nearly half of all patients with SOD1-ALS have no known family history of ALS3

The American Neurological Association recommends all persons with ALS be offered genetic testing and counseling4

NfL is a component of the axonal cytoskeleton found in the CSF and blood5

  • Multiple studies in ALS have shown that NfL levels are correlated with the rate of disability accrual and overall survival5
  • Plasma NfL is a sensitive biomarker of the underlying disease process in ALS5
  • As a result, plasma NfL is thought to be an important prognostic marker of disease progression in ALS5

CSF=cerebrospinal fluid; NfL=neurofilament light chain.

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INDICATION

QALSODY® (tofersen) is indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. This indication is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with QALSODY. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).

IMPORTANT SAFETY INFORMATION

Warnings and Precautions

Myelitis and/or Radiculitis

Serious adverse reactions of myelitis and radiculitis have been reported in patients treated with QALSODY. Six patients treated with QALSODY experienced myelitis or radiculitis in the clinical studies. Two patients discontinued treatment with QALSODY and required symptomatic management with full resolution of symptoms. In the remaining 4 patients, symptoms resolved without discontinuation of QALSODY. If symptoms consistent with myelitis or radiculitis develop, diagnostic workup and treatment should be initiated according to the standard of care. Management may require interruption or discontinuation of QALSODY.

Papilledema and Elevated Intracranial Pressure

Serious adverse reactions of papilledema and elevated intracranial pressure have been reported in patients treated with QALSODY. Four patients developed elevated intracranial pressure and/or papilledema. All patients received treatment with standard of care with resolution of symptoms, and no events led to discontinuation of QALSODY. If symptoms consistent with papilledema or elevated intracranial pressure develop, diagnostic workup and treatment should be initiated according to the standard of care.

Aseptic Meningitis

Serious adverse reactions of aseptic meningitis (also called chemical meningitis or drug-induced aseptic meningitis) have been reported in patients treated with QALSODY. One patient experienced a serious adverse reaction of chemical meningitis, which led to discontinuation of QALSODY. One patient experienced a serious adverse reaction of aseptic meningitis, which did not lead to discontinuation of QALSODY. In addition, nonserious adverse drug reactions of CSF white blood cell increased, and CSF protein increased have also been reported with QALSODY. If symptoms consistent with aseptic meningitis develop, diagnostic workup and treatment should be initiated according to the standard of care.

Adverse Reactions

The most common adverse reactions (≥10% of patients treated with QALSODY and greater than placebo) were pain, fatigue, arthralgia, cerebrospinal fluid white blood cell increased, and myalgia.

Please click for full Prescribing Information.

References: 1. QALSODY Prescribing Information, Cambridge, MA: Biogen. 2. Brown RH, Al-Chalabi A. Amyotrophic lateral sclerosis. N Engl J Med. 2017;377(2):162-172. 3. Chiò A, Mazzini L, D’Alfonso S, et al. The multistep hypothesis of ALS revisited: the role of genetic mutations. Neurology. 2018;91(7):e635-e642. 4. Roggenbuck J, Eubank BHF, Wright J, Harms MB, Kolb SJ; ALS Genetic Testing and Counseling Guidelines Expert Panel. Evidence-based consensus guidelines for ALS genetic testing and counseling. Ann Clin Transl Neurol. 2023;10(11):2074-2091. 5. Thompson AG, Gray E, Verber N, et al. Multicentre appraisal of amyotrophic lateral sclerosis biofluid biomarkers shows primacy of blood neurofilament light chain. Brain Commun. 2022;4(1):fcac029. 6. Heckler I, Venkataraman I. Phosphorylated neurofilament heavy chain: a potential diagnostic biomarker in amyotrophic lateral sclerosis. J Neurophysiol. 2022;127(3):737-745.

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