Qalsody

Qalsody (tofersen) is given by a healthcare provider as an intrathecal injection. The injection will take about 1 to 3 minutes. The medication is injected into your lower back directly into your spinal fluid. Intrathecal injections help Qalsody (tofersen) get directly to your brain because the fluid from your spine flows to your brain. This allows the medication to work directly in your nervous system to prevent the buildup of mutated SOD1, which has been linked to ALS.

Studying a new medication usually takes many years. Sometimes, a medication can be approved in a shorter amount of time because it fills a medical need that isn’t currently met by other medications already available in the market. When the FDA approves a medication under the Accelerated Approval Program, the decision is based on a “marker” that suggests a clinical benefit (e.g., a medical condition improving). But for the medication to receive full approval, the manufacturer must prove the medication’s clinical benefit through additional studies at a later time. Qalsody (tofersen) got an accelerated approval after early studies showed that it lowered blood levels of proteins called neurofilament light (NfL). The level of these neurofilament proteins are typically high when there's nerve damage. So, a lower NfL level could mean Qalsody (tofersen) helps to prevent nerve damage. But additional studies are needed to confirm whether having lower NfL levels actually slows the worsening of ALS.

In clinical studies, people who took Qalsody (tofersen) for 6.5 months had less physical symptoms of ALS, but so did the placebo group. It's important to note that the Qalsody (tofersen) group had much lower blood levels of neurofilament light (NfL), which are proteins that typically indicate nerve damage. A lower level of NfLs might mean that Qalsody (tofersen) can help prevent nerve damage and slow progression of ALS, but more studies are needed to confirm this.