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Study design

VALOR was a phase 3, 28-week, randomized, double-blind, placebo-controlled clinical study in participants with weakness attributable to ALS and a SOD1 mutation confirmed by a central laboratory (N=108)1,2

VALOR baseline characteristics

  • The prespecified primary analysis population (n=60, modified intent to treat [mITT]) had a slow vital capacity (SVC) ≥65% of predicted value and met prognostic enrichment criteria for rapid disease progression, defined based on their prerandomization ALSFRS-R decline slope and SOD1 mutation type1
  • The non-mITT population (n=48) had an SVC ≥50% of predicted value and did not meet the enrichment criteria for rapid disease progression1
  • Baseline disease characteristics in the overall ITT population (combined mITT and non-mITT population) were generally similar in patients treated with QALSODY and patients who received placebo, with slightly shorter time from symptom onset and higher plasma NfL at baseline in the QALSODY group1
  • Concomitant riluzole and/or edaravone use was permitted for patients. At baseline, 62% of patients were taking riluzole, and 8% of patients were taking edaravone1
  • Mean baseline ALSFRS-R score was 36.9 (5.9) in the QALSODY treatment group and 37.3 (5.8) in the placebo group1
  • Median time from symptom onset was 49.5 weeks in the QALSODY arm and 63.4 weeks in the placebo arm1
  • Mean age at study entry was 49.8 years (range: 23 to 78 years), and 57% of participants were male1

Primary endpoint

The primary endpoint was change from baseline to 28 weeks in the ALSFRS-R total score in the mITT population, analyzed using the joint rank test to account for mortality in conjunction with multiple imputation to account for missing data for withdrawals other than death.1

ALSFRS-R=Amyotrophic Lateral Sclerosis Functional Rating Scale–Revised; ITT=intent-to-treat.

At the end of VALOR, study participants had the option to enroll in the VALOR open-label extension (OLE)1

The interim VALOR OLE analysis at 52 weeks was designed to compare early- vs delayed-start QALSODY in the full ITT trial population.1

Adults aged 23 to 78 years (N=108) diagnosed with weakness attributable to ALS and a SOD1 mutation confirmed by a central laboratory were randomized 2:1 to receive treatment with either QALSODY 100 mg or placebo for 24 weeks (3 loading doses followed by 5 maintenance doses).1

The VALOR OLE is ongoing1

Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).

See the results of the VALOR trial

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. Miller TM, Cudkowicz ME, Genge A, et al; VALOR and OLE Working Group. Trial of antisense oligonucleotide tofersen for SOD1 ALS. N Engl J Med. 2022;387(12):1099-1110.

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