FDA advisory gives negative opinion on Hemispherx' Ampligen

The Arthritis Advisory Committee (AAC) of the US Food and Drug Administration last week voted against approval of the New Drug Application for US biotech firm Hemispherx Biopharma’s (NYSE: HEB) Ampligen (rintatolimod) for chronic fatigue syndrome (CFS).

Although the FDA is not obliged to follow advisory committee recommendation, the negative view effectively creates a major roadblock in the company’s decades-long pursuit of getting its experimental CFS treatment into the market. The FDA rejected that initial application in 2009, saying the company’s clinical studies “did not provide credible evidence of efficacy of Ampligen” in patients with CFS (The Pharma Letter May 29, 2009).

On the question: "Considering the totality of the data, is there substantial evidence of efficacy for Ampligen for the treatment of patients with chronic fatigue syndrome (CFS)?” the AAC voted 9 no, 4 yes and 1 AAC member left the room and did not vote.