The Food and Drug Administration on Tuesday authorized the first drug for a rare genetic form of the neurological disease ALS, despite uncertainty about the treatment’s effectiveness.
The decision reflects the agency’s push for greater flexibility in approving treatments for patients with devastating diseases and few, if any, options.
Biogen, the pharmaceutical company that markets the drug, said it would price the drug “in a range comparable to other recently launched treatments for ALS.” An ALS therapy approved last year cost $158,000 a year.
The drug, known scientifically as tofersen and which will be sold under the brand name Qalsody, targets a mutation in a gene known as SOD1 that is present in about 2% of the approximately 6,000 cases of ALS diagnosed in the States. United every year. Fewer than 500 people in the United States at any given time should be eligible.
The agency authorized the treatment via a policy that allows a drug to be fast-tracked to market in certain circumstances before there is conclusive evidence of its effectiveness. Biogen will need to provide confirmatory evidence, from ongoing clinical research, to keep the drug on the market.
The decision is the first conditional approval given to a drug for ALS, or amyotrophic lateral sclerosis, which typically causes paralysis and death within years. Less than half of patients eligible for Qalsody survive more than three years after their diagnosis.
The approval is based on evidence that the drug can significantly reduce levels of a protein that has been linked to nerve cell damage. Biogen argued that these results are reasonably likely to help patients, even though the drug, in a clinical trial, did not significantly slow disease progression, as measured by patients’ ability to speak. , swallowing and performing other activities of daily living. .
Despite uncertainty about its benefits, approval of Qalsody is widely seen as more justifiable than that of Aduhelm, another drug from Biogen. Aduhelm caused an uproar when the FDA approved it in 2021 to treat Alzheimer’s disease despite a lack of evidence that it worked.
At a meeting last month, a panel of independent FDA advisers unanimously recommended that the agency grant conditional approval of Qalsody, even though a majority of advisers concluded there was no compelling evidence of its effectiveness.
ALS patients and advocacy groups have mounted an impassioned campaign for the drug. FDA officials wrote last month that their approach to evaluating these drugs has been shaped by the agency’s “interactions with patients and their caregivers who describe their willingness to accept less certainty about the drug.” efficiency in exchange for faster access to essential drugs”.
Patients receive Qalsody as an injection into the spinal canal every few weeks. The drug was found to be generally safe, although a small number of patients developed spinal cord inflammation.
Before Qalsody, only three ALS drugs were approved in the United States and they did not significantly alter the course of the disease.