The U.S. Food and Drug Administration (FDA) gave Biogen an Accelerated Approval on April 25, 2023, for Qalsody^™ (tofersen). Licensed from Ionis, it is indicated to treat patients who have a rare form of amyotrophic lateral sclerosis (ALS) associated with mutations in the gene for superoxide dismutase 1 (SOD1). Delivered through intrathecal (into the spinal canal) injections, the recommended dose is 100mg. After three doses are given at 14-day intervals, maintenance doses are administered once every 28 days. The exact cost for Qalsody has not been revealed, but Biogen expects it to be within a “range compared to other recently launched” treatments for ALS. Shipments to healthcare providers are planned to begin next week, but access to treatment may be delayed while providers are trained to use it. Prescribing information is here.

At a Glance

• Brand (Generic) Name: Qalsody (tofersen)
• Manufacturer: Biogen
• Date Approved: April 25, 2023
• Indication: treatment for ALS in adults who have mutations in SOD1 genes
• Dosage Forms Available: single-dose vials of 100mg/15mL for intrathecal injection
• Launch Date: within one week
• Estimated Annual Cost: in line with other recently approved drugs for ALS
• ALS is a rapidly worsening neurological disease usually caused by random genetic mutations. It destroys the nerves that control voluntary movement, eventually resulting in paralysis and death within only a few years from diagnosis.
• Men, Caucasians, and individuals in their 60s may be slightly more likely to have ALS. Only about 2% of patients have a family history of the disease.
• Although as many as 32,000 Americans may have ALS, the Centers for Disease Control and Prevention (CDC) estimates that fewer than 500 patients have the SOD1 mutations that may be candidates for Qalsody use.
• Qalsody is an antisense drug that interferes with the production of SOD1, a protein associated with ALS. It also decreases blood levels of neurofilament light (NfL), a proxy biomarker for nerve degeneration.
• Patients receiving Qalsody in a clinical trial averaged 35% less SOD1 in their cerebrospinal fluid (CSF) after 28 weeks of treatment, compared to 2% less for patients given a placebo. Qalsody also produced an average 55% reduction in blood levels of NfL, compared to a 12% increase for those who were given a placebo. 
• Side effects from treatment were reported by about 4% more of patients (18.1%) receiving Qalsody than placebo (13.9%). They included fatigue, headaches, muscle and joint pain, and pain associated with administering the drug.
• Six other drugs are FDA approved for treating ALS. None is indicated for SOD1 ALS, however.

Because Accelerated Approval for Qalsody was based on interim results of clinical trials, additional positive results from more advanced trials will be needed before full FDA approval is granted. A designated Orphan Drug, it was approved under the FDA’s Fast Track and Priority Review paths.