December
24, 2012 (HealthDay News) A U.S. Food and Drug Administration
advisory committee on Thursday recommended against approval of the
first proposed drug to treat chronic fatigue syndrome (CFS),
intravenous Ampligen (rintatolimid).
Noting
that the FDA denied approval for Ampligen in November 2009 due to
methodological concerns, an advisory panel of the FDA reviewed a new
analysis of one of the trials submitted in 2009. In Thursday's 8-to-5
vote, the advisory panel said the company hadn't provided enough data
to support the approval of Ampligen. Although the FDA isn't bound to
follow the recommendations of its advisory committees, it usually
does so.
Ampligen,
an immunomodulatory double stranded RNA drug developed by Hemispherx
Biopharma in Philadelphia, PA, has been shown to help a subset of
patients with CFS by reducing symptoms. The drug needs to be infused
twice a week and is expensive, with a manufacturing cost of about
$1,000 per patient per month. Largely positive anecdotal reports were
presented in a teleconference conducted by the FDA with individuals
with CFS who had used the drug.
"Patients
with severely debilitating CFS suffer for years, both physically and
cognitively, with little to no quality of life," write the
authors of the Arthritis Advisory Committee Meeting briefing
document. "The impact is felt by their families and our nation
as a medical/social consequence. The current standard of care is
palliative and provides little to no permanent relief."