Qalsody can slow progression of SOD1-ALS, aid function: Study
Most of 16 patients given early access also viewed therapy favorably
Treatment with Qalsody (tofersen) substantially slowed disease progression and reduced markers of nerve damage in people with amyotrophic lateral sclerosis (ALS) associated with SOD1 mutations (SOD1-ALS) who took part in an expanded access program in Germany.
Patient-reported outcome measures also suggested a favorable perception of Qalsody, with most reporting an easing or stability in particularly bothersome symptoms, and most recommending the treatment.
“This clinical practice cohort added more data on clinical and patient-reported outcomes and neurofilament response during [Qalsody] treatment in SOD1-related ALS,” the researchers wrote.
The study, “Clinical and patient-reported outcomes and neurofilament response during tofersen treatment in SOD1-related ALS—A multicenter observational study over 18 months,” was published in the journal Muscle & Nerve.
Qalsody now is approved in the European Union and US to treat SOD1-ALS
Qalsody, marketed by Biogen, was conditionally approved in the U.S. in April 2023 to treat ALS due to SOD1 mutations, which are found in up to 20% of all with familial ALS and up to 2% of patients with the more common sporadic ALS.
The therapy also recently was approved in Europe under exceptional circumstances, a pathway for medicines treating unusually rare diseases. Before that, it was available to patients across Europe via expanded access programs or EAPs, which allow people with life-threatening conditions to access promising investigational products outside of clinical trials.
Researchers reported findings in 16 adults treated with Qalsody for at least six months under an EAP in Germany that opened in January 2022.
Patients had mean age of 53, and they had been living with ALS for a mean of slightly more than four years. A total of eight SOD1 mutations were identified across this group, and 10 patients had no family history of the disease.
Qalsody was given via monthly intrathecal (into-the-spinal canal) injections for a mean of 11 months, and it was “very well tolerated” with no discontinuations, the researchers wrote. The most commonly reported adverse events included headache, back pain, nausea, fever, and hair loss.
During treatment, 50% of the patients experienced either a stabilization (one adult) or an improvement (seven people) in their functional abilities, as measured with the ALS Functional Rating Scale-Revised (ALSFRS-R).
Disease progression was slower during treatment in half of the 16 patients and stable in the remaining eight. Lung function, as measured by slow vital capacity, remained stable.
Researchers also found that blood levels of neurofilament light chain (NfL), a marker of nerve damage, fell in all but one patient after Qalsody initiation, reducing by an average of 58%.
Ten of 14 patients reported an easing in a highly bothersome symptom
Participants were asked to report their treatment expectations and experiences using the Measure Yourself Medical Outcome Profile (MYMOP2), a patient-generated scale. Scores are arrived at by patients first prioritizing their two most bothersome symptoms or impairments, and selecting an activity important to them but made difficult by their condition. Next, they need to quantify the severity of their symptoms and affected activity on a seven-point scale.
Data was available for 14 patients, who most commonly prioritized walking and lower limb function, followed by arm or hand function, general mobility or autonomy, and breathing as domains with bothersome symptoms.
Ten (71%) were considered “responders” to Qalsody, meaning they perceived an improvement in at least one of their two target symptoms, with two responders reporting an easing in both symptoms. The other four patients were considered partial responders, with a perceived improved in one target symptom but a worsening in the other. Participants also generally reported a reduction in the impairment of their chosen activity.
Overall, high satisfaction was seen with the treatment, as determined by a mean score of 83 (out of a maximum of 100) on the nine-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) scale. In contrast, treatment usability and convenience were rated more modestly — a mean score of 50 — possibly owing to its intrathecal administration and monthly dosing, the scientists noted.
After six months of treatment, most patients (80%) recommended Qalsody, and this increased to 100% at both nine months and one year of use. These positive results “were remarkable as not all patients reported a stabilization or even improvements during treatment,” the researchers wrote.
“Further research is needed to better understand the individual differences of clinical and NfL treatment response, and the extent to which tofersen can modify the course of disease during long-term treatment,” they added.
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