Deja Vu?

“I just try and not think about it, because it gets me furious” multiple sclerosis patient

The story sounds eerily similar. Citing questions regarding study quality and side effects, the FDA refuses to approve a drug for a condition for which few or no effective drugs exist. After hearing story after story of patients who have been dramatically helped by the drug, the patient community goes ballistic. They send letters and emails and gather 10,000 signatures on a petition asking the FDA to change their decision.

advocating for ampligen

Astounded at the FDA’s rejection of a drug for MS, the patient community successfully fought back.

They argue they should be allowed to determine whether they want to accept the risk that comes with taking the drug.

It sounds very much like what played out in Ampligen and chronic fatigue syndrome (ME/CFS), but these event concerned a multiple sclerosis drug called Lemtrada.

Studies indicated the drug resulted in 50% fewer new attacks and reduced numbers of new lesions. The drug had already been approved in forty countries including Canada, Europe and the U.K. but the FDA said no go to it in the U.S. March of last year.

The FDA didn’t like the study design, though, and was concerned about the potentially dangerous, even fatal side-effects of the drug.

The chief advocate for the National MS Society said he didn’t have a clue what the FDA was thinking: “Trying to get a grip on what the FDA is thinking around this is quite puzzling and frustrating,” said Timothy Coetzee, chief of advocacy, services and research for the National MS Society.

The doctors on the ground couldn’t understand either as they were clearly seeing benefits. Seventy doctors using the drug in clinical studies signed a letter asking FDA to approve the drug.  They called it “an important tool” in their fight against the disease.

FDA ampligen chronic fatigue syndrome

Doctors using the drug were shocked at the FDA’s action

Some patients had had remarkable outcomes. One doctor who was headed for a wheelchair is able to walk and work full-time since he got an infusion in 2003. Another who would choke on his food and often needed a cane to walk is now able to ride his bike.

After Genzyme provided a re-analysis of its data, the FDA overturned its disapproval and approved the drug last December.


It’s hard not to think of Ampligen at a time like this. The doctors who have used it strongly promote it. Not everybody gets helped, but stories of major improvement and even recovery abound.  It’s maker, Hemispherx Biopharma, provided the FDA with a re-analysis of its data as well. Even more so than MS patients, people with ME/CFS don’t have many good drug options.

But after highlighting concerns about safety in a drug the doctors using it found to be very safe,  the FDA failed to approve Ampligen. This was despite the fact no other drugs have been in the FDA pipeline for ME/CFS for twenty years, and none are in sight now.  The FDA’s call for expensive new studies essentially removed Ampligen as an option for people with ME/CFS. A drug that was clearly safe and clearly helped many people hit the dust.

FDA chronic fatigue syndrome

After action from patients and doctors and a re-analysis of the data, the FDA approved the drug.

Since then the FDA has removed its major objection to approval – safety –  leaving efficacy as the last remaining stumbling block. Even the FDA panelists who voted against approval, however, stated Ampligen was clearly having an effect. They simply wanted more study.

The reasons for the FDA’s rejection of Ampligen are fading fast. There are ways to conditionally approve a drug which allow more information to be gathered about its effects without small drug companies like Hemispherx Biopharma having to pony up vast amounts of cash. Why that’s not being done is unclear.

With the multiple sclerosis community successful in their attempt to get the FDA approval for a drug with potentially very significant side effects, it’s getting harder and harder to see why Ampligen, a drug with no significant safety issues, is not receiving at least conditional approval from the FDA for chronic fatigue syndrome.

  • Next up – Advocates Request Congressional Hearing to Address Ampligen Rejection


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