Repritned with the kind permission of Cort Johnson and Health Rising
By Cort Johnson
Ampligen became the first drug approved for chronic fatigue syndrome (ME/CFS) anywhere in the world this week when ANMAT, the Argentine FDA, approved the drug for use in people with severe ME/CFS. Why should we get excited about Ampligen's approval in Argentina? Because Argentina has a modern medical system which includes having a rigorous drug approval process. Hopefully this will be a harbinger of things to come for Ampligen.
Argentina was the first Latin American country Hemispherx Biopharma (HB) – the maker of the drug – tried to get Ampligen approval in. The process, which took four years, began when HB teamed up with GP Pharm, an Argentine pharmaceutical company. GP Pharm used the same studies to shepherd the approval through ANMAT, the Argentine form of the FDA, that HB used in its FDA application for Ampligen two years ago.
Ampligen was approved for use in people with "severe ME/CFS" – a designation that was based on the type of ME/CFS patients that took part in the company's original U.S. studies. A person with severe ME/CFS in Argentina needs to meet the new IOM and the (old) Holmes criteria, and have a Karnovsky score between 40 and 60 in order to get access to the drug.
40: Disabled; requires special care and assistance.
50: Requires considerable assistance and frequent medical care.
60: Requires occasional assistance, but is able to care for most of his personal needs.
Hemispherx believes that at least 100,000 and perhaps as many as 500,000 patients in the 42 million person country will meet the criteria.
Equel's has been making the rounds of media outlets to discuss Ampligen. A financial analyst with Crystal Research agreed in an interview at Small Cap Nation – a financial media outlet focused on small companies – that the potential for Ampligen in the ME/CFS market alone was huge. With Ampligen the sole drug even being considered at this point for ME/CFS it would have the market to itself. He called the Argentine approval "very significant" and put the potential valuation of the drug in the multi-billion dollar range.
He also agreed the Argentine approval will make getting approval elsewhere easier.
The FDA Question
I talked with Nancy McGrory at Hemispherx about the recent approval. She believes the approval could help HB in other countries with similar regulatory drug processes. The fact that Ampligen passed muster in a country with a modern medical system with a rigorous drug approval process will likely, McGrory thought, prompt other countries to take a much closer look at the drug.
Ten years ago, she noted, most pharmaceutical drug companies concentrated on getting drug approval at the FDA first in the belief that making it through the FDA's notoriously tough regulatory process would give other countries confidence that the drug was safe and effective. The FDA – which is focused first on protecting the public health – has proved so risk averse lately, though, that some drug companies are choosing to get drug approval outside the U.S. first.
That brought up the question, though, what's going with FDA approval for Ampligen? When I asked McGrory about the reception Ampligen was getting at the FDA these days, she sounded hopeful. She said she believed that the work of patients, advocates and ME/CFS experts to educate the FDA about ME/CFS had paid off. The FDA Workshop and other meetings, she believed, really got the message across about how serious disease is, and that produced a sea-change in the agencies attitude towards ME/CFS. The FDA she thought, would like very much to approve a drug for this illness.
Thomas Equels, HB's new President has made Ampligen approval the companies top priority and has stated that he will to do whatever is necessary to get it done. McGrory said HB officials have meet with the FDA and the NIH several times, and will soon meet to finalize the protocol for what will hopefully be the final phase III study on ME/CFS.
The FDA wants, and is going to get a several hundred person trial. Equel's job now is to find partners to fund it. The companies new stance – that it’s committed to reach out and either license its technology or find a major pharmaceutical company or investor to assist it – is a distinct change of pace at HB.
In another hopeful note, Hemispherx reported it's at long last identified a subset of high responders to Ampligen and will present a paper to that effect at the IACFS/ME conference in October. Finding high responders to Ampligen would, of course, greatly help HB be successful in a phase III trial.
When asked in an extensive interview with Wall Street Transcript about what a potential investor should know about Ampligen Equels said:
"It is very important to me that they know that we have worked diligently, over a long period of time, to develop this experimental drug, Ampligen, because we believe it has a multifaceted important role to play in the future of medicine in ME/CFS, immunooncology, as a viral-vaccine enhancer, and as a broad-spectrum prophylactic and early-onset viral therapy.
At Hemispherx, our team has the dedication to accomplishing those goals in large part based upon the fact that we have a strong and unrelenting belief that we are doing something that is very important for people who suffer from these diseases, many with clearly unmet medical needs. We are going to do our best to work, with what we have, to accelerate the process — in part by acquiring partners — so that we accomplish those goals."
About the Author: Cort Johnson has had ME/CFS for over 30 years. The founder of Phoenix Rising and Health Rising, Cort has contributed hundreds of blogs on myalgic encephalomyelitis, chronic fatigue syndrome, fibromyalgia and their allied disorders over the past 10 years. Find more of Cort's and other bloggers' work at Health Rising.