Dr. Ferran J. García

A decision on Hemispherx BioPharma’s new drug application for Ampligen, twice delayed by the FDA earlier this year, is still in limbo.

William A. Carter, CEO of the Center City biotech company, said last week the company is responding to issues raised by the Food and Drug Administration during its review of Ampligen, an experimental treatment for chronic fatigue syndrome.

Hemispherx has spent more than $500 million and more than three decades developing the drug.

Carter last week provided an update on the status of the FDA’s review of Ampligen to BioMedReports, a Web site that provides information on life sciences firms.

“We continue to be in contact with the [FDA] concerning certain requests they have made over the last year that have to do with toxicology,” Carter said. “This is nonclinical work on the drug. We have continued to complete reports and we expect to sometime this quarter, the fourth quarter, complete a set of requirements [from the FDA] that have to do with preclinical toxicology.”

In its latest quarterly report filed with the Securities and Exchange Commission, Hemispherx noted it is working with the Lovelace Respiratory Research Institute in Albuquerque, N.M., on animal toxic studies in support of its Ampligen application.

The August filing noted the studies were requested by the FDA and that the studies were “substantially completed,” with summary reports issued to the agency during the third quarter.

“Data for final FDA reports are presently undergoing internal auditing at Lovelace and Hemispherx with a projected completion of the final report for late 2009 to early 2010,” the company stated in the filing.

Carter told BioMedReports the FDA has also “done a number of audits” of the company’s clinical sites and manufacturing facility during the past 12 months.

“The clinical inspections resulted in no findings that required [major] corrective action by the company,” he said, adding that the agency did note compliance issues at its facility in New Brunswick and at a contract facility in Spokane, Wash.

The manufacturing plant in Spokane is where Ampligen is packaged.

Carter said over this past summer the company addressed the FDA’s compliance issues at its New Jersey facility and submitted a report to the agency for review. He said the company expects to complete remediation efforts at the Spokane facility within the next several weeks.

“Until all those reports are completed and filed, the agency can withhold a final decision on the commercialization of the product,” said Carter.

Earlier this week, the company announced the discovery of a novel retrovirus in chronic fatigue syndrome patients.

A study published in the Oct. 8 issue of Science Express by a consortium of researchers found a new retrovirus in the blood cells of 67 percent of chronic fatigue syndrome patients and in 3.7 percent of healthy control subjects.

Hemispherx is collaborating with the Whittemore Peterson Institute, in Reno, Nev., to evaluate the potential role of Ampligen in the clinical treatment of chronic fatigue syndrome patients who have a specific immune deficiency in cell activity.

The company plans to provide an update on its programs targeting the disorder at a November Health and Human Services Chronic Fatigue Syndrome Advisory Committee meeting in Washington, D.C.