Guest guest Posted December 18, 2009 Report Share Posted December 18, 2009 Final Actions The FDA's decision whether to approve a new drug for marketing comes down to answering two questions: 1. Do the results of well-controlled studies provide substantial evidence of its effectiveness? 2. Do the results show that the product is safe under the explicit conditions of use in the proposed labeling? Here "safe" is a relative term; it means that the benefits of the drug appear to outweigh its risks. When the review is complete, the FDA writes to the applicant to say the drug is either approved for marketing, is "approvable," provided minor changes are made, or is not approvable because of major problems. In the last case, the applicant can then amend or withdraw the NDA or ask for a hearing. Once its NDA is approved, a drug is on the market as soon as the firm gets its production and distribution systems going. Fast Track, Accelerated Approval and Priority Review FDA This lady this blog describes the process best: it all has to do with Wall Street also... The FDA is sometimes the puppet of Wall Street and has let slip by all kind of medications and are worthless. Also look at the Physicians who write for medications and their interest in the companies. They are usually financial.. below are some research links WHAT'S IN THE FUTURE FOR PATIENTS WITH IPF Poor Choice of Primary Outcome in a Clinical Trial of Pirfenidone in Patients with IPF Click on he site bar for the full PDF file ATS 2009: Pirfenidone for Idiopathic Pulmonary Fibrosis Displays Contradictory Results in 2 Trials May 27, 2009 FDA Grants Orphan Drug Designation to Pirfenidone for Treatment Of Patients With Idiopathic Pulmonary Fibrosis March 24, 2004 basically NO ones to finance Pirfeidone. Erna Erna 1992 bird fanciers syndrome CAD & MI 2004 ILD 2008 NSIP 2009 Washington State To: Breath Support <Breathe-Support >Sent: Fri, December 18, 2009 8:52:17 AMSubject: Pirfenidone According to their press release, InterMune Inc announced that it submitted a New Drug Application (NDA) with the FDA, seeking approval to market Pirfenidone for the treatment of patients with IPF. Pirfenidone has been granted Orphan Drug and Fast Track designation by the FDA. InterMune has dedicated almost ten years to the development of new medicines for patients with IPF. This is the first NDA ever submitted to the FDA for a medicine to treat IPF patients. Preclinical and in-vitro evidence had shown that pirfenidone has both anti-fibrotic and anti-inflammatory effects. Results from three adequate well-controlled Phase 3 studies have shown evidence of a treatment effect in IPF patients and the compound has been safe and generally well tolerated. In Oct 2008 Pirfenidone was approved for use in IPF patients in Japan and is marketed as Pirespa by Shionogi in that country. [The Pulmonary Paper] My question is...when a NDA has been submitted how long does it take to get approval and market? C_53_Familial IPF_5/09, Washington HOPE doesn't disappoint! Quote Link to comment Share on other sites More sharing options...
Guest guest Posted December 18, 2009 Report Share Posted December 18, 2009 Thanks Bruce, When it says that perfenidone has anti-fibrotic effects, what does that mean? C_53_Familial IPF_5/09, Washington HOPE doesn't disappoint! To: Breathe-Support Sent: Fri, December 18, 2009 9:18:42 AMSubject: Re: Pirfenidone It can vary widely within a range from as little as 6 months to as muchas 3 years with the norm being 9 - 24 months from what I've found.Pirfenidone does have orphan drug status in their favor in the approvalprocess but also has the less than hoped for results of the trialsworking against them, especially failing to meet the goal in one of thetwo trials.The FDA had agreed to a fast-track process on Pirfenidone, whichtypically is six months or thereabouts. So, based on the applicationbeing submitted in November of this year, I would have expected ananswer by May, 2010. But the failure of Capacity 1 could complicatematters. Note that it took Intermune a much longer than anticipated timejust to put the application together after the completion of the trials.I still think a decision is likely sometime between May and September,2010, but thats not by any means set in stone.Now, to go off topic a bit. I'm not a Pirfenidone proponent so statingthis from a general rather than a specific situation. I find theapproval time from the point of application to the point of approval orrejection to be absurdly long. It seems to me that the requirementsshould be known and the documents submitted should either meet or failto meet those requirements. It doesn't take 2-3 years to review, butit's more a matter of backlog and of getting additional information.Those matters just shouldn't be there. If it fails to meet requirementsthen it should be rejected, not go back and forth for years. Let themresubmit at a later date if they can meet requirements. Similarly, if amed does meet requirements, then it shouldn't take so long to see thatif the application is properly prepared. I do realize the volume isextremely high. This timeframe has been extremely problematic as cancerdrugs are submitted, many hopeful of curing the disease, as opposed toPirfenidone which even at best is only hopeful of slowing a disease.Yes, the approval process is thorough to protect us, but thoroughdoesn't have to be slow.>> According to their press release, InterMune Inc announced that itsubmitted a New Drug Application (NDA) with the FDA, seeking approval tomarket Pirfenidone for the treatment of patients with IPF.. Pirfenidonehas been granted Orphan Drug and Fast Track designation by the FDA.InterMune has dedicated almost ten years to the development of newmedicines for patients with IPF. This is the first NDA ever submitted tothe FDA for a medicine to treat IPF patients.>> Preclinical and in-vitro evidence had shown that pirfenidone has bothanti-fibrotic and anti-inflammatory effects. Results from three adequatewell-controlled Phase 3 studies have shown evidence of a treatmenteffect in IPF patients and the compound has been safe and generally welltolerated. In Oct 2008 Pirfenidone was approved for use in IPF patientsin Japanand is marketed as Pirespa by Shionogi in that country. [ThePulmonary Paper]>> My question is...when a NDA has been submitted how long does it taketo get approval and market?>>> C_53_Familial IPF_5/09, Washington> HOPE doesn't disappoint!> Quote Link to comment Share on other sites More sharing options...
Guest guest Posted December 18, 2009 Report Share Posted December 18, 2009 First, understand that the statement was a press release from Intermune so it's not an independent statement. What Intermune would have you believe is that Pirfenidone slows the progression of the fibrosis, hence the anti-fibrotic effects. Whether that is a reasonable conclusion is debatable as the trials were far from overwhelming. One of the two trials did not conclude it had benefit. When you combine the two trials they seemed to indicate the possibility of some limited benefit in the first 36 weeks on a primarily older male group with extremely early stage IPF. At 72 weeks there was no appreciable difference between the drug and placebo groups. Pirfenidone will also point to the Japanese trial but that was a Phase 2 trial, not a Phase 3, and many think both the measurement and the early ending of that trial make any conclusions suspect. I guess time will ultimately tell. If it is approved, individuals will face another decision as to whether to deal with the side effects for the very limited probability it will benefit them. > > > > According to their press release, InterMune Inc announced that it > submitted a New Drug Application (NDA) with the FDA, seeking approval to > market Pirfenidone for the treatment of patients with IPF. Pirfenidone > has been granted Orphan Drug and Fast Track designation by the FDA. > InterMune has dedicated almost ten years to the development of new > medicines for patients with IPF. This is the first NDA ever submitted to > the FDA for a medicine to treat IPF patients. > > > > Preclinical and in-vitro evidence had shown that pirfenidone has both > anti-fibrotic and anti-inflammatory effects. Results from three adequate > well-controlled Phase 3 studies have shown evidence of a treatment > effect in IPF patients and the compound has been safe and generally well > tolerated. In Oct 2008 Pirfenidone was approved for use in IPF patients > in Japanand is marketed as Pirespa by Shionogi in that country. [The > Pulmonary Paper] > > > > My question is...when a NDA has been submitted how long does it take > to get approval and market? > > > > > > C_53_Familial IPF_5/09, Washington > > HOPE doesn't disappoint! > > > Quote Link to comment Share on other sites More sharing options...
Guest guest Posted December 19, 2009 Report Share Posted December 19, 2009 Thanks Erna...good information C_53_Familial IPF_5/09, Washington HOPE doesn't disappoint! To: Breathe-Support Sent: Fri, December 18, 2009 4:16:01 PMSubject: Re: Pirfenidone Final Actions The FDA's decision whether to approve a new drug for marketing comes down to answering two questions: 1. Do the results of well-controlled studies provide substantial evidence of its effectiveness? 2. Do the results show that the product is safe under the explicit conditions of use in the proposed labeling? Here "safe" is a relative term; it means that the benefits of the drug appear to outweigh its risks. When the review is complete, the FDA writes to the applicant to say the drug is either approved for marketing, is "approvable, " provided minor changes are made, or is not approvable because of major problems. In the last case, the applicant can then amend or withdraw the NDA or ask for a hearing. Once its NDA is approved, a drug is on the market as soon as the firm gets its production and distribution systems going. Fast Track, Accelerated Approval and Priority Review FDA This lady this blog describes the process best: it all has to do with Wall Street also... The FDA is sometimes the puppet of Wall Street and has let slip by all kind of medications and are worthless. Also look at the Physicians who write for medications and their interest in the companies. They are usually financial.. below are some research links WHAT'S IN THE FUTURE FOR PATIENTS WITH IPF Poor Choice of Primary Outcome in a Clinical Trial of Pirfenidone in Patients with IPF Click on he site bar for the full PDF file ATS 2009: Pirfenidone for Idiopathic Pulmonary Fibrosis Displays Contradictory Results in 2 Trials May 27, 2009 FDA Grants Orphan Drug Designation to Pirfenidone for Treatment Of Patients With Idiopathic Pulmonary Fibrosis March 24, 2004 basically NO ones to finance Pirfeidone. Erna Erna 1992 bird fanciers syndrome CAD & MI 2004 ILD 2008 NSIP 2009 Washington State From: worth <hope2thend (AT) yahoo (DOT) com>To: Breath Support <Breathe-Support@ yahoogroups.. com>Sent: Fri, December 18, 2009 8:52:17 AMSubject: Pirfenidone According to their press release, InterMune Inc announced that it submitted a New Drug Application (NDA) with the FDA, seeking approval to market Pirfenidone for the treatment of patients with IPF.. Pirfenidone has been granted Orphan Drug and Fast Track designation by the FDA. InterMune has dedicated almost ten years to the development of new medicines for patients with IPF. This is the first NDA ever submitted to the FDA for a medicine to treat IPF patients. Preclinical and in-vitro evidence had shown that pirfenidone has both anti-fibrotic and anti-inflammatory effects. Results from three adequate well-controlled Phase 3 studies have shown evidence of a treatment effect in IPF patients and the compound has been safe and generally well tolerated. In Oct 2008 Pirfenidone was approved for use in IPF patients in Japan and is marketed as Pirespa by Shionogi in that country. [The Pulmonary Paper] My question is...when a NDA has been submitted how long does it take to get approval and market? C_53_Familial IPF_5/09, Washington HOPE doesn't disappoint! Quote Link to comment Share on other sites More sharing options...
Guest guest Posted January 14, 2010 Report Share Posted January 14, 2010 Pat, The FDA is expected to make it's decision by May of this year. Please be a bit cautious in your own expectations for this drug. The trials that have been done show it may be of some value in slowing the progression in some people with IPF/UIP. It does not 'stop fibrosis' and even Intermune (the manufacturer) doesn't claim that it does that. That said if one has IPF/UIP it may very well be worth a try assuming it's approved. It has not been tested and Intermune is not seeking approval for it's use for any other ILD than IPF/UIP. Beth Moderator Fibrotic NSIP 06/06 Dermatomyositis 11/08 To: Breathe-Support Sent: Thu, January 14, 2010 12:16:31 PMSubject: Pirfenidone Hi Everyone, Do any of you happen to know when we can expect pirfenidone to be approved by the FDA? Or how to find that information. It was approved in Japan 10-16-08, and is supposed to be a great medication in stopping fibrosis. Maybe if we all sent messages to the FDA they would move faster. Pat Quote Link to comment Share on other sites More sharing options...
Guest guest Posted January 14, 2010 Report Share Posted January 14, 2010 The review date for approval is May. As to saying its a great medication in stopping fibrosis there is no evidence to that effect. Thats way overstating any of the trials. The approval in Japan was based on very little except that there is nothing else. It may have some benefit to some people but to lead anyone to believe it cures the disease somehow isn't appropriate. There is plenty of information available including the results of the US trials is available in the Files section on this site. > > Hi Everyone, > Do any of you happen to know when we can expect pirfenidone to be > approved by the FDA? Or how to find that information. It was approved > in Japan 10-16-08, and is supposed to be a great medication in stopping > fibrosis. Maybe if we all sent messages to the FDA they would move > faster. > Pat > Quote Link to comment Share on other sites More sharing options...
Guest guest Posted January 14, 2010 Report Share Posted January 14, 2010 Pat, My thoughts on Pirfenidone are such that, when at Tulane in December, I totally forgot to ask Dr. Lasky about his thoughts on Pirfenidone. Pirfenidone, as Bruce and Beth said, only minamally slow (if even that) the progression of this disease. Intermune is in the process of gearing up for the promotion of this drug to the health industry. I have no doubt that your pulmonologist and mine will be visited by sales reps from Intermune, who will try and put the best face on it. But I have no intention of changing my medications. However I will keep exercising and eating well to live for the day when a better treatment is found for us. I do believe that will happen. Jerry/Mississippi/54/IPF/dx April 05 Who believes that hard times help us appreciate the good times even more. Quote Link to comment Share on other sites More sharing options...
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