Food and Drug Administration on Tuesday first drug approved For a rare genetic form of neuropathic ALS, despite uncertainties about treatment efficacy.
The decision reflects the agency’s commitment to increasing flexibility in approving treatments for patients with devastating diseases who have few, if any, options.
Biogen, the drug company that brought the drug to market, said it would price the drug “within a range comparable to other recently launched treatments for ALS.” His ALS drug approved last year cost $158,000 a year.
Scientifically known as tofersen, the drug, which will be marketed under the brand name Qalsody, targets mutations in a gene known as SOD1 that is present in approximately 2% of the approximately 6,000 cases of ALS diagnosed each year in the United States. targeted. less than 500 Expected to be eligible in the US at any time.
Agency approved treatments via policies that allow drugs to be brought to market faster under certain circumstances Before there was conclusive proof that it works. Biogen needs to provide corroborative evidence from ongoing clinical studies to keep medicines on the market.
The decision is the first conditional approval given to a drug to treat ALS, or amyotrophic lateral sclerosis, which commonly causes paralysis and death within a few years. Fewer than half of patients eligible for Qalsody survive three years or more after diagnosis.
This approval is based on evidence that the drug can significantly reduce levels of proteins associated with nerve cell damage. Biogen claims that these results are likely to help patients. measured by The patient’s ability to speak, swallow, and perform other activities of daily living.
Despite uncertainty about its benefits, Qalsody’s approval is widely seen as more legitimate than that of Aduhelm, another of Biogen’s drugs. Aduhelm sparked protests when the FDA approved it to treat Alzheimer’s disease in 2021, despite a lack of evidence that it works.
At a meeting last month, the FDA’s panel of independent advisers unanimously recommended that the FDA grant Qalsody conditional approval.
ALS patients and advocacy groups have launched a passionate campaign against the drug. FDA officials I have written Last month, their approach to evaluating such medicines was dictated by patients and their peers who said they were willing to accept uncertainty about efficacy in exchange for earlier access to the agency’s “badly needed medicines.” It was formed by “interactions with caregivers.”
Patients receive Qalsody as an injection into the spinal canal every few weeks. A few patients developed spinal cord inflammation, but the drug was found to be generally safe.
Prior to Qalsody, only three drugs for ALS were approved in the United States and did not significantly alter the course of the disease.
