Tiny biotech warns patients it is running out of time 

Following FDA’s denial of a request to reconsider a complete response letter, Stealth Biotherapeutics and the patients who see its investigational drug elamipretide as their only hope are making last-ditch appeals to FDA Commissioner Marty Makary.

The company’s roller-coaster regulatory journey seemed to be nearing resolution last year when the Cardiovascular and Renal Drugs Advisory Committee voted 10-6 that Stealth had demonstrated elamipretide’s efficacy. That vote came after extraordinary interventions by CDER Director Patrizia Cavazzoni and OND Director Peter Stein, who offered alternative interpretations of the data that could support approval, departing sharply from FDA reviewers, who argued that the evidence fell short...