FDA approves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene

✓Supports this

“Some trials are open only to people who have a particular biological or genetic characteristic.”

Describes an ALS trial that did this. Everyone in it had to have a fault in the SOD1 gene, confirmed by a laboratory test, before they could take part, and the treatment it led to is licensed only for people with that same gene fault.

The effectiveness of Qalsody was evaluated in a 28-week, randomized, double-blind, placebo-controlled clinical study in 147 patients with weakness attributable to ALS and a SOD-1 mutation confirmed by a central laboratory.

Effectiveness

iAdds context

“Markers measured in blood or spinal fluid that may reflect nerve-cell damage are being explored for tracking ALS/MND and are used in trials.”

One concrete example rather than the basis for the whole sentence. It covers only the blood form of the marker and only its use in a trial, and says nothing about spinal fluid or about tracking the condition outside a trial.

The approval was based on a reduction in plasma neurofilament light (NfL), a blood-based biomarker of axonal (nerve) injury and neurodegeneration.

Action

iAdds context

“A biomarker gives researchers a measurable signal of whether a treatment is having an effect, which can let a trial show that in less time and in fewer people.”

Not the basis for the sentence, but it shows what this looks like in practice. The US regulator approved an ALS treatment on the strength of a change in a biomarker rather than a change in how people were, on the understanding that the change is reasonably likely to predict a real benefit.

Qalsody is approved under the accelerated approval pathway, under which FDA may approve drugs for serious conditions where there is an unmet medical need and a drug is shown to have an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients.

Effectiveness

iAdds context

“For some genetic changes, knowing your result can open access to particular clinical trials or gene-targeted treatments.”

One worked example rather than a general picture. The US regulator approved a treatment only for ALS caused by a fault in the SOD1 gene, and the trial running to confirm its benefit is open only to people who carry that gene change. That is roughly 2% of ALS, and it is a United States decision that says nothing about what is available elsewhere.

To confirm the clinical benefit of Qalsody, a Phase 3 randomized, double-blind, placebo-controlled trial is ongoing in individuals who are carriers of the SOD1 genetic mutation who do not yet have symptoms.

Effectiveness