FDA skeptical of benefits from experimental ALS drug

The Food and Drug Administration has issued a negative review of a closely watched experimental drug for the debilitating illness known as Lou Gehrig’s disease

WASHINGTON -- Federal health regulators issued a negative review Monday 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 Wednesday, a panel of FDA advisers will take a non-binding vote on whether the drug warrants approval.

The meeting is being closely watched as an indicator of the FDA’s approach to experimental drugs with imperfect data and its ability to withstand outside pressure.