GOP Sen. Johnson investigating FDA rare disease drug rejections

WASHINGTON — Sen. Ron Johnson (R-Wis.) said he is investigating the Food and Drug Administration’s rejections of rare disease treatments.

The Wisconsin Republican is requesting denial letters, called complete response letters, that the FDA sent to makers of rare disease treatments for conditions including ataxia and Sanfilippo syndrome. He said he’s heard that some of the issues in those letters are “nitpicky things.” 

“We’re going to find out exactly what issues the FDA listed for their ‘nos’,” he said.