December 20, 2012, 6:52 p.m. ET
Panel Rejects Drug for Chronic Fatigue Syndrome
By JENNIFER CORBETT DOOREN A federal advisory panel on Thursday said a
proposed medicine from Hemispherx Biopharma Inc. HEB -5.26% to treat
chronic fatigue syndrome isn't ready for approval, dealing another
setback to the drug maker.
Hemispherx Biopharma is seeking approval from the U.S. Food and Drug
Administration for the drug, Ampligen. The product, which been in
development for more than two decades, was reviewed Thursday by the
agency's arthritis-drugs advisory panel, which is made up of non-FDA
medical experts. In an 8-to-5 vote, the panel said the company didn't
provide sufficient data to support the approval of Ampligen. The vote
amounts to a recommendation that FDA not approve the drug.
Several panel members said they struggled with their decisions because
it appears the drug works in certain patients even if it wasn't
strongly shown in the clinical data presented to the panel.
"I think the advisory panel in general hopes there will be an
effective drug and hopes this might be the drug" for chronic fatigue
syndrome, said Lenore Buckley, the panel's chairwoman and a professor
at Yale School of Medicine. "We are interested in seeing more data." A
decision by the FDA on whether to approve Ampligen is expected by
Almost three dozen patients or family members testified in person or
via video before panel, urging them to approve Ampligen. Anita Kathryn
Patton, who's had chronic fatigue syndrome for more than 20 years, was
first given Ampligen in 1997.
"It was like rising from the dead," Ms. Patton said of her disease
improvement. She and some other patients are receiving Ampligen
through a special access program at a cost of about $25,000 a year.
At issue is whether Ampligen, which is the drug's proposed brand name,
is effective and safe. The FDA said there was missing data in clinical
studies that made it hard to tell whether the drug is safe. Potential
safety concerns include infections and liver problems. FDA medical
reviewers questioned whether the data meet drug-approval requirements
demonstrating "substantial evidence" of safety and efficacy, or
Theresa Michele, an FDA team leader, said the agency believes that
chronic fatigue syndrome is a serious disease that needs treatments,
but said clinical studies submitted in support of products have to
meet federal drug approval standards showing "substantial" safety and
effectiveness. "We have to be certain a drug works and we clearly know
what the risks are," Dr. Michele said.
The FDA and the company differed on whether one of the main clinical
studies reached a goal showing a statistically significant test
showing patients receiving the drug were able to walk on a treadmill
for a longer period than patients receiving placebo injections.
The company said on average there was an improvement of about one
minute while the FDA said the difference appeared to be about 20
"Regardless of the [measurement] approach there's a consistent pattern
of benefit in the data, which favor Ampligen," said William Carter,
Hemispherx chief executive. At a minimum, Dr. Carter said, the drug
"prevents further disease progression." After the meeting, Dr. Carter
said he had no comment.
Chronic fatigue syndrome, also known as myalgic encephalomyelitis, is
believed to affect more than one million Americans, according to the
Centers for Disease Control and Prevention. The condition is marked by
severe fatigue, muscle pain and memory and concentration problems. It
isn't known what causes the condition and there are currently no
Ampligen, an injectable drug, is believed to boost the body's immune
system and fight viruses.
Write to Jennifer Corbett Dooren at firstname.lastname@example.org