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Hemispherx awaits FDA decision on Ampligen NDA for chronic fatigue syndrome - "The Pink Sheet Daily"

Hemispherx BioPharma (AMEX:HEB) was recently featured in an article in the "The Pink Sheet Daily", which outlines the boost in drug development efforts from the FDA for chronic fatigue syndrome (CFS), and discusses the progress of the company's Ampligen drug - a potential first approved treatment for the disease. 


Hemispherx BioPharma (AMEX:HEB) was recently featured in an article in the "The Pink Sheet Daily", which outlines the boost in drug development efforts from the FDA for chronic fatigue syndrome (CFS), and discusses the progress of the company's Ampligen drug - a potential first approved treatment for the disease. 

The writer of the piece, Sue Sutter, says that the FDA has embarked on a drug development initiative for chronic fatigue syndrome and myalgic encephalomyelitis, and at the same time it is considering whether Hemispherx’s Ampligen should be the first drug approved for CFS.

The agency is planning a series of activities with patients, advocacy groups and other stakeholders focused on development of drugs to treat the symptoms of CFS/ME. 

These actions are aimed at exploring the burden of disease and its impact on quality of life, quantitative outcome measures or endpoints that determine if symptoms improve with intervention, and how drug efficacy should be clinically tested based upon these endpoints or measurements, the FDA announced on its website.

"The initiative is a major step in FDA’s efforts to raise the profile of CFS as a drug development target," Sutter writes. 

The agency suggested an accelerated approval route as an option, Sutter continues, and in doing so, "appears to be following the directive laid out in the FDA Safety and Innovation Act (FDASIA), which was signed into law on July 9."

In addition to reauthorizing PDUFA, FDASIA included provisions aimed at encouraging broader use of regulatory mechanisms, such as accelerated approval and fast track designation, to speed the approval of drugs for serious and life-threatening conditions.

Cue Hemispherx. The company, amid this regulatory environment, is on the cusp of a potential breakthrough as it sits on what could potentially be the first recognized commercial treatment for CFS – with just months away from anticipated FDA approval. 

Hemispherx’s resubmitted new drug application for Ampligen is currently under review by the Division of Pulmonary, Allergy and Rheumatology Products, with a February 2, 2013 deadline date. The company has said it expects an FDA advisory committee meeting on the application on December 20.

Ampligen, which has been in development for about 20 years, has faced a series of regulatory challenges, including a 2009 complete response letter in which the FDA requested an additional clinical trial. 

But in July, Hemispherx announced that the FDA had reversed its previous decision, agreeing to review new analyses of a previously completed phase III trial - in lieu of requiring an additional trial. 

This is in part because in January 2011, the Center for Drug Evaluation and Research announced plans to consolidate oversight of CFS treatments in the Division of Pulmonary, Allergy and Rheumatology Products, whereas previously, CFS applications were assigned to at least six different review divisions. 

In a recently filed 10Q report, Hemispherx said that Ampligen, which has orphan drug status, represents the first drug in the class of large RNA (nucleic acid) molecules to apply for NDA review.

Nucleic acid compounds represent a potential new class of pharmaceutical products that are designed to act at the molecular level for the treatment of human diseases. RNA is a group of naturally occurring informational molecules which orchestrate a cell’s behavior which, in turn, regulate the action of cells that compromise the body’s immune system.

The drug, which is a 30-minute infusion into the bloodstream, works by stimulating the immune system through a receptor called TLR-3. 

Clinical trials of Ampligen already conducted by the company include studies for the potential treatment of CFS/ME, Hepatitis B, HIV and cancer patients. 

"All of these potential uses will require additional clinical trials to generate the safety and effectiveness data necessary to support regulatory approval," it said. Over 1,000 patients have participated in the Ampligen clinical trials representing the administration of more than 90,000 doses of this drug.

According to the Center for Disease Control, there are around 4 million people affected by CFS in the US, costing the nation around $9.1 billion per year.  The illness, which has an unknown etiology thought in part to be related to immune dysfunction, causes profound fatigue and other debilitating symptoms, with much of the impacted population bedridden. 

In March of this year, a peer reviewed analysis of data from the a trial of Ampligen was published showing that the proportions of Ampligen patients with exercise improvements of at least 25% and at least 50% respectively, were 1.7 and 1.9-fold greater than those patients on placebo, Hemispherx said. 

Along with this data, the new analyses contained in the resubmitted filing included an examination of quality-of-life parameters in patients who either met or failed the primary endpoint of exercise treadmill testing.

The NDA also included an analysis showing that compared to those taking placebo, patients on Ampligen were able to reduce their use of concomitant medications that may prolong the QT interval. Prolongation of the QT interval is a risk factor for arrhythmia and sudden cardiac death.

In a statement announcing the resubmission, Hemispherx said:  “Hemispherx believes that the data and analyses provided in its complete response may be relevant to the potential for approval of Ampligen under this expanded statutory authority. 

“There can be no assurance, however, how the FDA will implement the new FDASIA provisions.”

Hemispherx also owns Alferon N Injection, which is an injectable formulation of natural alpha interferon, which was approved by the FDA in 1989 for the treatment of certain categories of genital warts - one of the main culprits of cervical cancer.  

Commercial sales of Alferon were halted in March 2008 when the company's finished goods inventory expired, and since then, it has been working to build a bio reactor manufacturing facility with a 600 litre tank, investing around $8 million to expand its capacity. The FDA is expected to visit and inspect the facility soon, with sales anticipated to start following this sometime in 2013. 

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