The Food and Drug Administration (FDA) has approved Biogen's Qalsody, the first medication for a rare genetic form of the neurological disorder A.L.S. (amyotrophic lateral sclerosis). The drug targets a mutation in a gene known as SOD1 that is present in only about 2% of the roughly 6,000 cases of A.L.S. diagnosed in the United States each year, and fewer than 500 people in the United States at any given time are expected to be eligible. Patients will receive Qalsody as an injection into the spinal canal every few weeks. The drug was found to be generally safe, though a small number of patients developed inflammation of the spinal cord.
The decision to approve the medication was based on evidence that the drug can significantly reduce levels of a protein that has been linked to damage to nerve cells. Biogen has argued that these results are reasonably likely to help patients, even though the drug, in a clinical trial, did not significantly slow the progression of the disease, as measured by patients’ ability to speak, swallow and perform other activities of daily living.
The FDA authorized the treatment via a policy that allows a drug to be fast-tracked onto the market under certain circumstances before there is conclusive proof that it works. Biogen will be required to provide confirmatory evidence, from ongoing clinical research, to keep the drug on the market.
The approval of Qalsody reflects the FDA's push to be more flexible in approving treatments for patients with devastating illnesses and few, if any, options. Despite the uncertainty about its benefit, Qalsody's approval is widely seen as more justifiable than that of Aduhelm, another drug from Biogen. Aduhelm prompted an outcry when the FDA approved it in 2021 to treat Alzheimer’s despite a lack of evidence that it worked.
Before Qalsody, only three A.L.S. medications were approved in the United States, and they have not significantly altered the course of the disease. Biogen said it would price the drug “within a range comparable to other recently launched A.L.S. treatments.” An A.L.S. therapy approved last year was priced at $158,000 annually.
A.L.S. patients and advocacy groups mounted an impassioned campaign for the drug, and FDA officials wrote last month that their approach to evaluating such medications had been shaped by the agency’s “interactions with patients and their caregivers who describe their willingness to accept less certainty about effectiveness in return for earlier access to much-needed medicines.”