The Food and Drug Administration on Tuesday approved the first drug for a rare genetic form of the neurological disorder ALS, despite uncertainty about the treatment’s effectiveness.
The decision reflects the agency’s push for more flexibility in approving treatments for patients with devastating diseases and few or no options.
Biogen, the pharmaceutical company that markets the drug, said it would keep the price of the drug “in a range similar to other recently launched ALS treatments.” ALS therapy approved last year cost $158,000 a year.
The drug, which is scientifically known as tofersen and will be sold under the brand name Qalsody, targets a mutation in a gene known as SOD1, which is present in about 2 percent of the roughly 6,000 ALS cases diagnosed each year in the United States. the United States are diagnosed. Fewer than 500 people in the United States are expected to qualify at any given time.
The agency approved the treatment through a policy that allows a drug to be brought to market quickly under certain circumstances before there is conclusive evidence that it works. Biogen must provide confirmatory evidence from ongoing clinical trials to keep the drug on the market.
The decision is the first conditional approval granted for a drug against ALS, or amyotrophic lateral sclerosis, which generally causes paralysis and death within a few 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 associated with nerve cell damage. Biogen has argued that these results will reasonably help patients, even though the drug did not significantly slow disease progression in a clinical trial as measured by patients’ ability to speak, swallow, and perform other daily activities. feed. .
Despite the uncertainty about its benefit, the approval of Qalsody is widely seen as more warranted than that of Aduhelm, another Biogen drug. Aduhelm sparked outrage 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 advisers to the FDA unanimously recommended that the agency grant Qalsody conditional approval, though a majority of the advisers concluded there was no convincing evidence that it was effective.
ALS patients and advocacy groups passionately campaigned for the drug. FDA officials wrote last month that their approach to evaluating such drugs was shaped by the agency’s “interactions with patients and their healthcare providers that describe their willingness to accept less certainty about effectiveness in exchange for earlier access to much-needed drugs.” .”
Patients receive Qalsody by 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 have not significantly changed the course of the disease.