The FDA has granted conditional approval to a novel ALS medication, a move that will likely impact how other experimental medicines for the nerve-destroying illness are examined and assessed.
Tofersen, as previously known, is solely for ALS patients with a specific genetic abnormality. According to FDA estimates, this group accounts for less than 500 of the approximately 30,000 individuals in the United States who have the condition.
The medicines that were approved by the FDA recently were granted approval based on the medicine’s ability to increase the lives of patients. Tofersen, now sold as Qalsody, is different from others. It failed the pivotal clinical trial designed to show that it can halt the functional deterioration associated with ALS, or amyotrophic lateral sclerosis.
Tofersen’s clearance was based on its potential to reduce neurofilament light chain levels. It’s the first ALS medicine authorized based on “biomarker” evidence, establishing a precedent that might enable another road to market for certain companies. Tofersen is an antisense treatment developed jointly by Biogen and Ionis Pharmaceuticals. Tofersen inhibits the synthesis of SOD1, a protein thought to cause ALS when mutated.
The trial which helped in the FDA approval found that the medicine performs as per requirement, with patients who got it reporting considerably higher decreases in SOD1 than those who received placebos. Neurofilament light chain levels in treated individuals were significantly lower as well. According to research, high amounts of this protein in the fluid around the brain and spinal cord indicate nerve injury, most likely noticed in ALS patients.
Despite a successful trial, towers were still unable to meet the study’s primary purpose. Biogen met with FDA representatives many times after discovering this in 2021 to discuss the future steps for its medicine. The FDA says it urged the business to continue developing tofersen but warned that obtaining complete approval based on a single failed experiment would be difficult.
By the spring of 2022, Biogen had chosen to pursue “accelerated approval,” a sort of marketing clearance granted to medications that the FDA considers are reasonably expected to benefit patients according to their impact on disease indicators. The FDA agreed to study Biogen’s application and initially stated that it would provide a decision by January 25, 2023.
Patient advocacy groups are not satisfied with the FDA’s approval, arguing that it is not working hard to make more possible ALS medicines available to patients. They’ve emphasized how the FDA has granted fast clearance to drugs with a mixed track record that targets other components of the nervous system.
In light of this, the FDA organized a committee of independent experts in late March to examine Tofersen. The members agreed that the drug’s effect on the neurofilament light chain would likely predict clinical improvement.
Biogen stated that the medication will be priced competitively keeping in view the pricing of its competition. Relyvrio, which was authorized for ALS last year, costs around $158,000 per year, while Radicava, an oral form of an older branded drug, costs about $170,000 per year.
Tofersen is authorized for a small subset of ALS patients with the SOD1 gene. Given the small population, experts do not see Tofersen becoming a key product for Biogen. Experts believe the medicine will produce roughly $150 million in yearly sales.
Biogen anticipates that the patient’s out-of-pocket payments will be less than $50 per month.