Approved under FDA accelerated approval pathway. See Indication.
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Binding to SOD1 mRNA
Degrading SOD1 mRNA
Resulting in a reduction of SOD1 protein synthesis
SOD1=superoxide dismutase 1
Total CSF SOD1, an indirect measure of target engagement, and NfL, a blood-based biomarker of axonal injury and neurodegeneration, were
evaluated in VALOR.
VALOR ITT population Week 28 reduction in CSF SOD1 protein (geometric mean ratio to baseline)1
Difference in geometric mean ratios for QALSODY to placebo: 34%; nominal p<0.0001
VALOR ITT population Week 28 change in NfL
(geometric mean ratio to baseline)1
Difference in geometric mean ratios for QALSODY to placebo: 60%; nominal p<0.0001
At the maximum approved recommended dosing regimen, QALSODY does not prolong the QTc interval to any clinically relevant extent.
Baseline disease characteristics in the ITT population (combined modified intent-to-treat [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 group.1
*Concomitant riluzole and/or edaravone use was permitted for patients.
ALSFRS-R=Amyotrophic Lateral Sclerosis Functional Rating Scale–Revised; NfL=neurofilament light.
The mITT population (n=60):
Patients treated with QALSODY experienced less decline from baseline in the ALSFRS-R compared to placebo but the results were not statistically significant (QALSODY-placebo adjusted mean difference [95% CI]: 1.2 [-3.2, 5.5]). Other clinical secondary outcomes also did not reach statistical significance.
Note 1: N is the number of patients with baseline value.
Note 2: MI was used for missing data. Model included treatment, use of riluzole or edaravone, relevant baseline score and post-baseline values (natural log transformed data). Separate models for mITT and nonmITT were used and combined for ITT analyses.
Note 3: Adjusted geometric mean ratios to baseline, treatment differences in adjusted geometric mean ratios to baseline and corresponding 95% CIs and nominal p-values were obtained from the ANCOVA model for change from baseline including treatment as a fixed effect and adjusting for the following covariates: baseline disease duration since symptom onset, relevant baseline score, and use of riluzole or edaravone. The analysis was based on natural log transformed data.
Plasma NfL adjusted geometric mean ratio to baseline values in VALOR by study week for the ITT population
After completion of Study 1, patients had the option to enroll in an open-label extension study. At an interim analysis at 52 weeks, reductions in NfL were seen in patients previously receiving placebo who initiated QALSODY in the open-label extension study, similar to the reductions seen in patients treated with QALSODY in Study 1.
Earlier initiation of QALSODY compared to placebo/delayed initiation of QALSODY was associated with trends for reduction in decline on ALSFRS-R, SVC percent-predicted, and hand-held dynamometry (HHD) megascore that were not statistically significant.
Through all open-label follow-up at the time of the interim analysis, earlier initiation of QALSODY was also associated with a trend towards reduction of the risk of death or permanent ventilation, although it was not statistically significant.
These exploratory analyses should be interpreted with caution given the limitations of data collected outside of a controlled study, which may be subject to confounding.
Warnings and Precautions1
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.
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.
The most common adverse reactions (≥ 10% of patients treated with QALSODY and greater than placebo) were pain, fatigue, arthralgia, CSF white blood cell increased, and myalgia.
Less Common Adverse Reactions
Serious adverse reactions of myelitis and radiculitis; papilledema and elevated intracranial pressure; and aseptic meningitis have occurred in patients treated with QALSODY.
In the long-term extension study, nonserious adverse reactions of pyrexia have occurred with repeat administration of QALSODY.
*Pain includes preferred terms of pain, back pain, and pain in extremity.
†CSF white blood cell increased includes preferred terms of CSF white blood cell increased and pleocytosis.
QALSODY should be administered intrathecally using a lumbar puncture by healthcare professionals experienced in performing lumbar punctures.1
If the second loading dose is missed, administer QALSODY as soon as possible, and give the third loading dose 14 days later.
If the third loading dose is missed, administer QALSODY as soon as possible, and give the next dose 28 days later.
If a maintenance dose is missed, administer QALSODY as soon as possible, and give the next dose 28 days later.
Use aseptic technique when preparing and administering QALSODY intrathecally
Please click for full Prescribing Information for complete preparation and administration details.
Vials of QALSODY should be stored refrigerated, at a temperature between 2°C to 8°C (36°F to 46°F) in the original carton to protect from light. Do not freeze.1
If no refrigeration is available, QALSODY may be stored in its original carton, protected from light at or below 30°C (86°F) for up to 14 days.1
If removed from the original carton, unopened vials of QALSODY can be removed from and returned to the refrigerator, if necessary, for not more than 6 hours per day at or below 30°C (86°F) for a maximum of 6 days (36 hours).1
Please click for full Prescribing Information for complete preparation, storage, and handling details.
QALSODY is given through an intrathecal injection and should be administered at a treatment center by or under the direction of a healthcare professional experienced in performing lumbar punctures.
Please note that new centers are added regularly and may not be in this locator tool yet. For additional treatment centers, please call Biogen Support Services at 1-877-725-7639, Monday-Friday, 8:30 AM to 8:00 PM ET.
Biogen does not receive payment for this service and does not endorse or recommend treatment facilities that have chosen to be listed. Further, Biogen does not have oversight of and is not responsible for the actions of any treatment center.
Biogen does not guarantee availability at any particular treatment center. Every individual is unique, and only after careful medical evaluation can a course of treatment be determined.
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Answering general disease and product questions, as well as questions about Biogen Patient Support Services
Assisting with access to treatment and removal of nonclinical barriers
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