The FDA Returns to Its Bad Habits

Drug treatments for debilitating diseases are advancing rapidly, but the Food and Drug Administration isn’t moving with as much alacrity. Witness how the agency has slow-rolled its review of a breakthrough treatment for a rare degenerative nervous system disorder.

Friedreich’s Ataxia (FA) stems from a genetic mutation that causes a deficiency of the protein frataxin, which is critical to metabolic function. The progressive disease usually develops in children and results in difficulty walking, slurred speech, hearing and vision loss and shortness of breath. About 5,000 patients in the U.S. are afflicted with the disease, and most die in their 30s of heart problems. There is no approved treatment, only medications to manage symptoms such as diabetes.