Federal health regulators issued a negative review [March 28] of a closely watched experimental drug for the debilitating illness known as Lou Gehrig’s disease, after months of lobbying by patient advocates urging approval.
The drug from Amylyx Pharmaceuticals has become a rallying cause for patients with the deadly neurodegenerative disease ALS, their families and members of Congress who’ve joined in pushing the Food and Drug Administration to greenlight the drug.
But regulators said in a review that the company’s small study was “not persuasive,” due to missing data, errors in enrolling patients and other problems. On [March 30], a panel of FDA advisers will take a non-binding vote on whether the drug should be approved.
Amylyx’s drug is a combination of two older drugs: a prescription medication for liver disorders and a dietary supplement associated with traditional Chinese medicine. Amylyx has patented the combination in a powder and says the chemicals help protect cells from early death.
But the reviewers found the drug had “only a modest” effect on slowing the disease’s progression in a 137-patient, mid-stage study, which reviewers said was plagued with implementation and analysis problems. Typically, FDA approval requires two large studies or one study with a “very persuasive” effect on survival.
