What's a CRL?
CFS and Ampligen: A Creative Solution from the FDA?
- Thread starter JohnnyD
- Start date
Complete Response Letter.
http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/LawsActsandRules/ucm084143.htm
Oh - found it on google - it's 'Complete Response Letter' and is issued by the FDA when they're not approving a drug and tells them what changes need to be made before it can be approved.
Here's more detail on CRLs:
http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/LawsActsandRules/ucm084143.htm
I'm more than a bit baffled that Hemispherx would leave Bob Miller still on his hunger strike if they've known the FDA's decision since Friday.
Looking forward to getting a proper public statement one way or the other...
Nico
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Hi Laurel - I asked that! See post #22 above.
SEC? US Securities and Exchange Commission? Financial stuff, not medical/research?
Have I mis-googled???
Ember
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I think that you're being overly optimistic. Dr. Unger is committed to initiating a dialogue that will result in a broad umbrella definition with severity scales:
In November 2011, she described three phases in creating the new definition, i.e., data collection, the selection of severity scales and dialogue:
However in October 2012, Dr. Belay predicted that the data wouldn't be evaluated until the fall of this year:
Here is Dr. Belay's comment on the delay from June 2012:
It isn't clear from Dr. Belay's comments in October what phase of the study is expected to be completed within a year.
https://www.bioworld.com/content/fda-adds-ampligen-experts-roster-cfs-workshop-0
FDA Adds Ampligen Experts to Roster for CFS Workshop
By Randy Osborne
Staff Writer
The FDA published a notice in the Federal Register about the public workshop called "Drug Development for Chronic Fatigue Syndrome [CFS] and Myalgic Encephalomyelitis [ME]," slated for April 25-26 in Bethesda, Md.
It's the first such workshop held as part of the Patient-Focused Drug Development initiative, mandated in the authorization of the Prescription Drug User Fee Act, and a prime topic, given the uproar over the agency's refusal to approve Ampligen (rintatolimod) after a negative advisory panel meeting in December. (See BioWorld Today, Dec. 21, 2012.)
Ampligen was the only potential therapy on the horizon for CFS, long derided as "not a real disease," and many patients protested the FDA's inaction, especially those who had been benefiting from Ampligen by way of clinical trials conducted by its sponsor, Philadelphia-based Hemispherx Bioscience Inc. One activist patient, Robert Miller, went on a hunger strike to call attention to the issue. (See BioWorld Today, Feb. 6, 2013.)
Over the weekend, Miller wrote to the Health and Human Services' (HHS) secretary, Kathleen Sebelius, and to Margaret Hamburg, commissioner of the FDA, as well as others, calling the advisory panel meeting that turned thumbs-down on Ampligen "a pony show," and asking that he, as well as Daniel Peterson, who headed Hemispherx's trials and collaborated on others, be invited to the workshop.
"Did we not learn anything from HIV/AIDS?" asked Miller, who has been a CFS patient for 30 years and gained results with Ampligen. "Yes, [patients] know you are doing more, but after 30 years of nearly nothing, a Band-Aid appears huge. Every year, there are excuses they [HHS and its agencies] cannot do more."
In the Federal Register posting Monday, the FDA requested that anyone who wants to register to attend and/or take part in discussions submit their requests to the agency by April 8. The workshop will take up two main topics: disease symptoms and daily impacts that matter most to patients, and patients' perspectives on current approaches to treating CFS and ME.
Meanwhile, although the full roster of those invited to the workshop has not been made public, Kimberly McCleary, president and CEO of the Chronic Fatigue and Immune Dysfunction Syndrome Association of America, said she has seen a partial list.
"It's an interesting blend of CFS experts, so I think that will make [Miller] and others happy to see that, including two people who have been involved in the Ampligen studies," she told BioWorld Today. "They're both very capable spokespeople for what it's like to live with this. One is a physician herself who had to retire after she became ill. The core group [of those who formally present at the workshop] will be very compelling."
Peterson is not on the roster McCleary has seen. "One person contacted me independently and told me that he was going to be on the list," she added.
That's Bernard Munos, a consultant formerly with Eli Lilly and Co., of Indianapolis.
A CFS expert "who has looked at other treatments and done some pilot studies using other drugs and drug combinations" is included in the lineup, too, McCleary said.
The post in the Federal Register said the FDA "will try to accommodate all participants who wish to speak on [the first day, which is an afternoon session only], either through the panel discussions, audience participation, or the open public comment period," but warned that "the duration of comments may be limited by time constraints," probably anticipating a flood.
McCleary said "the bulk of the time, I would imagine, is going to be spent with the people the FDA has asked to attend," and each of those has been asked to stay for both days of the workshop, "so that they can then have a dialogue about the meeting in its entirety."
Hemispherx, which got its complete response letter from the FDA in February, said its hoped-for end-of-review conference with the agency will serve a precursor to submitting a formal appeal to the Office of New Drugs in the FDA's Center for Drug Evaluation and Research.
Ampligen, a Toll-like receptor 3 modulator, once was slated to undergo a Phase III trial to follow the AMP516 study, but the strategy later became to submit a new analysis of existing data from the 516, which used saline as its placebo control. But the decision to use saline was made in the early 1990s, before researchers began to use saline as a therapy for orthostatic and blood-volume problems in CFS. This meant the results may have been confounded by using a comparator with clinical efficacy, McCleary noted. (See BioWorld Today, Feb. 11, 2013.)
FDA Adds Ampligen Experts to Roster for CFS Workshop
By Randy Osborne
Staff Writer
The FDA published a notice in the Federal Register about the public workshop called "Drug Development for Chronic Fatigue Syndrome [CFS] and Myalgic Encephalomyelitis [ME]," slated for April 25-26 in Bethesda, Md.
It's the first such workshop held as part of the Patient-Focused Drug Development initiative, mandated in the authorization of the Prescription Drug User Fee Act, and a prime topic, given the uproar over the agency's refusal to approve Ampligen (rintatolimod) after a negative advisory panel meeting in December. (See BioWorld Today, Dec. 21, 2012.)
Ampligen was the only potential therapy on the horizon for CFS, long derided as "not a real disease," and many patients protested the FDA's inaction, especially those who had been benefiting from Ampligen by way of clinical trials conducted by its sponsor, Philadelphia-based Hemispherx Bioscience Inc. One activist patient, Robert Miller, went on a hunger strike to call attention to the issue. (See BioWorld Today, Feb. 6, 2013.)
Over the weekend, Miller wrote to the Health and Human Services' (HHS) secretary, Kathleen Sebelius, and to Margaret Hamburg, commissioner of the FDA, as well as others, calling the advisory panel meeting that turned thumbs-down on Ampligen "a pony show," and asking that he, as well as Daniel Peterson, who headed Hemispherx's trials and collaborated on others, be invited to the workshop.
"Did we not learn anything from HIV/AIDS?" asked Miller, who has been a CFS patient for 30 years and gained results with Ampligen. "Yes, [patients] know you are doing more, but after 30 years of nearly nothing, a Band-Aid appears huge. Every year, there are excuses they [HHS and its agencies] cannot do more."
In the Federal Register posting Monday, the FDA requested that anyone who wants to register to attend and/or take part in discussions submit their requests to the agency by April 8. The workshop will take up two main topics: disease symptoms and daily impacts that matter most to patients, and patients' perspectives on current approaches to treating CFS and ME.
Meanwhile, although the full roster of those invited to the workshop has not been made public, Kimberly McCleary, president and CEO of the Chronic Fatigue and Immune Dysfunction Syndrome Association of America, said she has seen a partial list.
"It's an interesting blend of CFS experts, so I think that will make [Miller] and others happy to see that, including two people who have been involved in the Ampligen studies," she told BioWorld Today. "They're both very capable spokespeople for what it's like to live with this. One is a physician herself who had to retire after she became ill. The core group [of those who formally present at the workshop] will be very compelling."
Peterson is not on the roster McCleary has seen. "One person contacted me independently and told me that he was going to be on the list," she added.
That's Bernard Munos, a consultant formerly with Eli Lilly and Co., of Indianapolis.
A CFS expert "who has looked at other treatments and done some pilot studies using other drugs and drug combinations" is included in the lineup, too, McCleary said.
The post in the Federal Register said the FDA "will try to accommodate all participants who wish to speak on [the first day, which is an afternoon session only], either through the panel discussions, audience participation, or the open public comment period," but warned that "the duration of comments may be limited by time constraints," probably anticipating a flood.
McCleary said "the bulk of the time, I would imagine, is going to be spent with the people the FDA has asked to attend," and each of those has been asked to stay for both days of the workshop, "so that they can then have a dialogue about the meeting in its entirety."
Hemispherx, which got its complete response letter from the FDA in February, said its hoped-for end-of-review conference with the agency will serve a precursor to submitting a formal appeal to the Office of New Drugs in the FDA's Center for Drug Evaluation and Research.
Ampligen, a Toll-like receptor 3 modulator, once was slated to undergo a Phase III trial to follow the AMP516 study, but the strategy later became to submit a new analysis of existing data from the 516, which used saline as its placebo control. But the decision to use saline was made in the early 1990s, before researchers began to use saline as a therapy for orthostatic and blood-volume problems in CFS. This meant the results may have been confounded by using a comparator with clinical efficacy, McCleary noted. (See BioWorld Today, Feb. 11, 2013.)
Ember
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JohnnyD -- perhaps you could help us understand this transaction:
"In this regard, we have registered $150,000,000 of securities for public sale pursuant to a universal shelf registration statement and we have been selling shares under this shelf registration statement and the New Maxim EDA. Through December 31, 2012, we had sold an aggregate of approximately 29,500,000 shares under this EDA."
...
"On November 26, 2012, all of the members of the Compensation Committee authorized the payment of bonus for the Company stock sold through the Maxim ATM based on the contractual obligation and opinion of independent counsel. For the year ending 2012, compensation was granted or paid related to the Executive Performance Incentive Program, as set forth in Section 3(c)(ii) of their respective Employment Agreements, for approximately $1,159,000 to each Dr. Carter and Mr. Equels, respectively."
From this page:
http://finance.yahoo.com/mbview/thr...8fa5-a446-4e5f-a4fa-7a23a9e9479c&tls=la,d,7,3
???
"In this regard, we have registered $150,000,000 of securities for public sale pursuant to a universal shelf registration statement and we have been selling shares under this shelf registration statement and the New Maxim EDA. Through December 31, 2012, we had sold an aggregate of approximately 29,500,000 shares under this EDA."
...
"On November 26, 2012, all of the members of the Compensation Committee authorized the payment of bonus for the Company stock sold through the Maxim ATM based on the contractual obligation and opinion of independent counsel. For the year ending 2012, compensation was granted or paid related to the Executive Performance Incentive Program, as set forth in Section 3(c)(ii) of their respective Employment Agreements, for approximately $1,159,000 to each Dr. Carter and Mr. Equels, respectively."
From this page:
http://finance.yahoo.com/mbview/thr...8fa5-a446-4e5f-a4fa-7a23a9e9479c&tls=la,d,7,3
???