WASHINGTON — Lawmakers pressed a top Food and Drug Administration official Thursday for answers on why the agency has balked at approving medicines to treat the fatal neurodegenerative condition ALS. Two key politicians even called out specific drug candidates by name, a rare congressional boost for the biotechs behind them.
The questions came during a hearing on neurodegenerative diseases hosted by the House Energy and Commerce committee that featured testimony from the National Institutes of Health, as well as patients and caregivers for conditions like Huntington’s disease and Alzheimer’s. But the lion’s share of questions were directed to the FDA’s drug center chief, Patrizia Cavazzoni. And most were about ALS.