Nadat de FDA, de Amerikaanse organisatie die geneesmiddelen goedkeurt, al eerder voor oponthoud had gezorgd, is er opnieuw vertraging voor het geneesmiddel Ampligen voor de behandeling van ME/CVS.

De eerste vertraging was het gevolg van aanvullende vragen van het FDA over het middel aan de producent. Als reden voor de nieuwe vertraging wordt de grote drukte van de autoriteit aangevoerd.

Het uitstel zal volgens de FDA drie maanden bedragen. Na de zeer lange ontwikkeltermijn moeten de patiënten dus voor de zoveelste keer nog wat geduld opbrengen.

Nieuwe klinische data over Ampligen worden gepresenteerd op de IACFS Conference in Reno, Nevada, 13 maart 2009.

The Long Wait Of Hemispherx’s Ampligen

For Hemispherx Biopharma Inc., it has been a long hard ride down — with its stock now having fallen to a low of $0.36 from its 52-week high of $1.20. Having been a lightning rod for controversy about its investigational chronic fatigue syndrome drug Ampligen, the company has been in the limelight more often for the wrong reasons in the past.

Chronic fatigue syndrome, or CFS, is a condition in which patients have persistent or relapsing fatigue. There is neither a known cause, nor an effective treatment for CFS.

Ampligen, which has been pacing the sidelines for nearly four decades, was expected by investors and patients to be approved by the FDA at least this month. But the FDA, which was expected to decide whether or not to approve Ampligen for chronic fatigue syndrome on February 25, has now extended the review period by another three months. Will the long wait prove fruitful for Hemispherx?

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Closing Thoughts

Hemispherx will be presenting new data on Ampligen at the upcoming International Association of Chronic Fatigue Syndrome in Reno, Nevada on March 13. The company has persevered through trying times with the hope of getting Ampligen approved by the FDA as a legitimate treatment for chronic fatigue syndrome. Will the decades-long effort of the company bear fruit at least this time? Stay tuned….

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Bron: TradingMarkets.com, 20-02-2009

FDA Extends Hemispherx’s NDA Review Date for Ampligen(r) as Potential Treatment for CFS

Extended User Fee Goal is Now May 25, 2009

PHILADELPHIA, Feb. 18, 2009 (GLOBE NEWSWIRE) — Hemispherx Biopharma, Inc. (AMEX:HEB) has received a letter from the Food and Drug Agency (“FDA”) indicating that the originally scheduled Prescription Drug User Fee Act (“PDUFA”) date on the Ampligen(r) (Poly I: Poly C12U) New Drug Application (NDA) would be extended by three months “in order to provide time for a full review of the submission.” Additional data were received by the FDA within 3 months of the user fee goal date.

Due to constraints at the FDA, specifically and including the increased workload related to the recently enacted and implemented FDA Amendments Act (“FDAAA”) and FDA’s Safety First/Safe Use initiatives, work priorities may change resulting in the Agency going past the customary PDUFA goal set for reviews of an application.

A decision was originally expected by February 25, 2009, for the Company’s submission of its Ampligen(r) NDA, which is designated as an Orphan Drug for the treatment of Chronic Fatigue Syndrome, which has no FDA approved treatments on the market. Ampligen(r) is also authorized for Emergency (compassionate) Cost Recovery Sales Authorization by the FDA and has a “promising” designation by the Agency on Health Research Quality (AHRQ): “Ampligen(r), an investigational drug that is not approved by the FDA, given intravenously to severely debilitated patients, yielded the most promising results.” The extended user fee goal date is now May 25th, 2009.

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Bron: TradingMarkets.com, 18-02-2009

Hemispherx Biopharma to Present New Ampligen(r) Clinical Data At the 9th International IACFS/ME Conference

Clinical/Lab Parameters of Potential Special Import to CFS Disease Severity

PHILADELPHIA, Feb. 18, 2009 (GLOBE NEWSWIRE) — Hemispherx Biopharma, Inc. (AMEX:HEB) announced its medical director, Dr. David Strayer, will present new data on Ampligen(r) (Poly I: Poly C12U) at the upcoming International Association of Chronic Fatigue Syndrome (IACFS/ME) in Reno, NV March 13, 2009. The mission of IACFS/ME is to promote, stimulate and coordinate the exchange of ideas related to CFS/ME research, patient care and treatment.

The oral presentation will include new data from the ongoing treatment IND/compassionate study (AMP 511) as well as the completed well-controlled pivotal Phase III study (AMP 516). Ampligen(r), an investigational stage therapeutic, has a pending New Drug Application (NDA) filed with the Food and Drug Administration (FDA) with an extended user fee goal of May 25, 2009.

Individuals suffering with CFS are known to be at greater risk than age-matched healthy populations with respect to certain catastrophic events including cancer, sudden cardio-vascular death and suicidal ideation. The new data will evaluate the potential of Ampligen(r), an experimental therapeutic, to mitigate certain of these events. Data utilized will integrate serially performed immunologic lab panels and EKGs, as well as physical performance scores and medical records (including concomitant medications used to alleviate certain symptoms of CFS).

Ampligen(r), an experimental product, may modulate nature’s primary gateway to immune response, mediated by receptors termed “toll-like receptors” (TLRs). Many other immuno-modulators may work downstream from the TLRs immune “gateways” at so-called “checkpoints”. Since “checkpoints” are not normally part of nature’s own immune-defense surveillance apparatus, these alternative “checkpoint” strategies may be associated with unintended consequences including significant toxicities.

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Bron: TradingMarkets.com, 18-02-2009

FDA delays Hemispherx drug vote

Hemispherx BioPharma is going to have to wait a little longer for a Food and Drug Administration decision on its lead new drug candidate — a therapeutic compound the company has had under development for more than three decades.

The FDA, citing an increased workload, said this week it is extending its review of Ampligen for another three months. A decision had been expected by Feb. 25.

Ampligen had been studied as a potential treatment for diseases ranging from AIDS and cancer to the avian flu.

The Center City biotech company is seeking approval to market Ampligen to treat chronic fatigue syndrome.

Hemispherx’s journey to getting a new drug candidate under review by the FDA has included: the company’s CEO, who co-invented Ampligen, being fired and then rehired; a contentious battle with a Wall Street short-seller and an alleged hostile takeover attempt by a group of South African businessmen.

Kim McCleary, president and CEO of the CFIDS (Chronic Fatigue and Immune Dysfunction Syndrome) Association of America, said her group is cautiously optimistic the FDA will find it has enough data to make a positive decision on Hemispherx’s application.

“If it is approved, it is expected to be quite expensive,” McCleary said. “[Ampligen] has shown to be effective in certain subsets of the condition. … If they get an approval, we think other companies will follow them in and it will lead to more research.”

McCleary noted no product has received FDA approval for treating chronic fatigue syndrome, a condition the association estimates impairs about 500,000 adults in the United States.

“Hemispherx has certainly had a wild ride through the [drug development] process,” she said.

Hemispherx was originally operated as HEM, a Maryland-based contract services company that performed mainly research work for the National Institutes of Health and NASA.

The company moved to Philadelphia in the early 1980s after Dr. William Carter, a former researcher at Johns Hopkins University in Baltimore and Hahnemann University Hospital in Philadelphia, reorganized Hemispherx into a drug-development company. Its focus became testing an experimental compound called Ampligen as a treatment for AIDS, cancer, chronic hepatitis and, most recently, chronic fatigue syndrome.

Carter did not return repeated calls for an interview for this story.

Hemispherx plans to present new data on Ampligen at a medical conference next month.

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Bron: Philadelphia Business Journal, 20-02-2009

Intro overgenomen van De Steungroep.