NEWSBRIEF: We now have an update on the FDA's much-delayed decision on Ampligen for chronic fatigue syndrome that explains why we've been kept waiting for so long.
William Carter, CEO of Ampligen-manufacturer Hemispherx Biopharma Inc., told BioMedReports that his company has been responding to FDA requests for further information about Ampligen's toxicology. According to it's last quarterly report, filed in August, Hemispherx has been working with another laboratory on animal toxicity studies and expects to get a final report to the FDA by late 2009 or early 2010.
Carter also said the FDA discovered "compliance issues" at the company's New Brunswick facility and a the facility Hemispherx contracted with for packaging. He said the FDA can withhold approval until those issues are rectified.
This isn't the news we've been hoping for, but at least now we know: approval is not coming this fall, as Carter had previously said. Since the FDA will have to re-audit the 2 facilities in question and then review the new toxicology data (once it's submitted), it seems like it would take a miracle to have a decision by the end of the year. Personally, I'd be surprised if we heard anything before spring.
In related news, Carter says Hemispherx is collaborating with the Whittemore Peterson Institute, which recently announced the discovery of a retroviral link in chronic fatigue syndrome, to see if Ampligen could be an effective treatment for XMRV.
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OK, here’s my take as an MBA/health care professional with ME/CFS. I have great (OK, almost unbridled) optimism for Ampligen’s imminent availability. Consider the following:
1) THE POTENTIAL MARKET SIZE. The implication that 4% of normal controls might have XMRV, represents a HUGE initial figure. Yes, the results need to be validated. And there will undoubtedly be subsets of XAND. BUT from the eyes of an investor, this is a HOT, HOT, HOT prospect.
2) THE SYNERGY BETWEEN XMRV RESEARCH AND EXISTING RESEARCH: “XMRV leads to immune dysfunction by damaging an antiviral pathway called RNase L. Ampligen, an immunostimulant drug currently waiting for FDA approval for treatment of CFS/ME works by treating this deficiency.” (http://chronic-fatigue-syndrome.suite101.com/article.cfm/cfsme_and_retroviruses ). I.e. we are NOT starting from square 1, and Hallelujia for that. Ampligen might not prove to be the BEST, and of course not the only treatment, but it is an appropriate first step towards comprehensive care for XAND.
3) THE RELATIVE SIMPLICITY OF XMRV AS A RETROVIRUS. If I recall correctly, it was Dr Silverman – XMRV researcher extraordinaire, who if I recall correctly has managed to be on the scientific SWAT teams for both the XMRV/malignant prostate work, as well as the XMRV/chronic fatigue work, who described XMRV as being like a “stripped down” version of a retrovirus. We have the AIDS community to thank for the masses of research on the highly complex HIV/AIDS virus. Not wanting to diminish the scope of the work ahead, but we have a HUGE head-start therefore with XMRV.
4) THE PRESSURE THE FDA WILL BE UNDER TO FAST-TRACK APPROVAL. A good barometer of the sense of urgency the NIH, FDA, pharma industry, etc. will be under is aptly reflected in the following blog excerpt: “Almost 4% of the general population tested positive for this retrovirus as well. These findings concerned the National Cancer Institute enough to call a closed door meeting held in July to discuss the public health implications of an XMRV infection… “NCI [National Cancer Institute] is responding like it did in the early days of HIV,” says Stuart Le Grice, head of the Center of Excellence in HIV/AIDS and cancer virology at NCI and one of the organizers of the July workshop. (http://cfswarrior.blogspot.com/2009/10/chronic-fatigue-syndrome-is-over-xmrv.html ). In short, even if one looks at ONLY the economic drivers, I suspect that the shareholders, board, executive, and staff of HEMISPHERx BioPharma, Inc. will be feverishly (pardon the pun) militating to pass all necessary hurdles asap, and get this product into the U.S. market ASAP! It’s a no-brainer for all involved to ramp up the sense of urgency – not just because it’s the right thing to do for a patient group that has been vilified for decades, but because there’s a massive market AND patient win here. And no, I’m not affiliated with any of the blogs or Hemispherx folks! Just one of the millions of ME/CFS patients – or should I say XAND patients – dying for more comprehensive care!
Maybe I’m off my rocker, but I really don’t think we need to exhort the FDA, researchers, and pharma industry to ignite the after-burners! shift to WARP speed!
More lame excuses from Dr. Carter. Truthfully, how can anyone believe anything the man says.
It is amazing to me that some people do. I have been sick since 1986 and following Hemispherx, in the company’s various forms since the 1990’s. You know what I think of the company.
When did Carter say they probably wouldn’t get a decision this year? Did you listen to the Maxin CC? I heard the presenter say ‘decision expected any day.’ That was Sept 29th.
Guide Response: The quarterly report said the toxicology data would go to the FDA late this year or early next year, and Carter told BioMedReports about the compliance issues at the two locations. I can’t see how the FDA could issue a decision before its requested information is turned in and reviewed, and it can withhold the decision until the two facilities are re-evaluated.