The agency’s trend towards more discretionary approval of medicines for patients with life-threatening conditions and few viable choices is reflected in this judgement.
The drug’s manufacturer, Biogen, has said that it plans to price the medication “within a range comparable to other recently launched A.L.S. treatments.” The yearly cost of an ALs treatment that was authorised only last year was $158,000.
Tofersen, marketed under the trade name Qalsody, targets a mutation in the SOD1 gene that accounts for around 2% of the 6,000 new instances of A.L.S. identified annually in the United States. It is estimated that less than 500 persons in the United States meet the requirements at any one moment.
The medication was approved by the government under a mechanism that enables drugs to be rushed to market in certain cases without definitive evidence of their efficacy. Biogen will need to show that the medicine is safe and effective in further clinical trials in order to keep it on the market.
This is the first time a medicine for amyotrophic lateral sclerosis (ALS), which often results in paralysis and death within a few years, has been awarded conditional approval. Only about half of Qalsody patients who are eligible for the drug actually make it that long.
Evidence showing the medicine can considerably lower levels of a protein associated to damage to nerve cells was used to justify the drug’s approval. Although the medicine did not substantially delay the course of the illness in a clinical study, Biogen has maintained that these findings are fairly likely to aid patients as evaluated by their capacity to talk, swallow, and conduct other activities of daily life.
Qalsody’s approval was considered as more defensible than that of another Biogen medicine, Aduhelm, despite the fact that its benefit is less clear. The F.D.A.’s 2021 approval of Aduhelm to treat Alzheimer’s disease despite a lack of proof that it works caused an uproar.
Qalsody was unanimously recommended for conditional clearance by a panel of independent advisors to the F.D.A. during a meeting last month, despite the fact that the majority of the panel agreed that there was not compelling evidence that it was effective.
Patients with A.L.S. and their supporters rallied passionately to get the medicine approved. In a recent blog post, F.D.A. officials explained how “interactions with patients and their carers who describe their willingness to accept less certainty about effectiveness in exchange for earlier access to much-needed medicines” had informed the agency’s approach to reviewing such therapies.
Qalsody is administered through spinal injection every several weeks. The medicine was determined to be harmless for the most part, while it did cause inflammation of the spinal cord in a few people.
Only three A.L.S. drugs had been licenced in the United States prior to Qalsody, and they had not made a major difference in the disease’s progression.