U.S. Food and Drug Administration Approves Cayston(R) (Aztreonam for Inhalation Solution) for the Improvement of Respiratory Symptoms in Cystic Fibrosis Patients with Pseudomonas Aeruginosa

http://www.gilead.com/pr_1393831

For all my buddies who couldn’t qualify for the study or the ‘compassionate care’ program, it’s your time now!!!

(now for the fight for insurance coverage… fun fun!)

Here’s the comment:

Dear Salty:

I think you’re being a little unfair to the FDA. They made a decision the CF community didn’t like — but that doesn’t mean they’ve been unethical as you suggest. And “foot dragging” would have meant no decision at all.

Still, I share your irritation because nobody — not even the CF Foundation — seems inclined to to explain exactly why the FDA decided that further clinical study was necessary. It always gets my dander up when the experts won’t let the regular folks in on the secrets, especially when their work is of such importance to CF patients like yourself.

The decision about which you complain was actually made last September and, as best I can tell, did not result in all access to inhaled astreonam being cut off. From the CFF web site:

“Because the FDA did not raise any safety issues related to the drug in their ruling, Gilead [the manufacturer] will continue to provide early access to aztreonam lysine for inhalation for those CF patients with limited treatment options and a serious risk of disease progression. Individuals with CF should contact their care centers to determine their eligibility to participate in the EAP.”

Presumably you’ve done that . . .?

My reply:

Although I appreciate your comment, it appears you aren’t completely up to speed on the study I speak of. The FDA has made clear their reasoning… the new board is not up to speed (pardon the pun) with the needs of cystic fibrosis patients, nor understands the need of getting drugs to market quickly to help save people’s lives.  As far as “foot dragging” I don’t know exactly how to address that.  The drug was up for review, they asked for more study results, Gilead studied, got more results, appealed the decision, the FDA now asks for another study, which will be reviewed at yet a later date… despite ”the FDA did not raise any safety issues related to the drug in their ruling” as you quoted.  I don’t know if there are better words than to say they are dragging their feet …as I have stated before, numbers are my game, not words so I apologise for my lack of vocabulary. …Yet this entire time  If there are no safety issues are related to the drug, then why on earth are they not approving it? I just don’t get it, and I guess that is what I was trying to describe in my previous post.

The inability to receive proper treatment for one month, may shave YEARS off a cystic’s life, no exaggeration there my dear. Speed means lives being saved. An ounce of prevention is worth a pound of cure my mother always told me. A good solution now is better than the best solution ten years from now.

For example, the year previous to my start on inhaled aztreonam I suffered horribly from lung infections, three rounds of IV antibiotics, at least three weeks in each round. And guess what drug I was infusing in my veins????? – AZTREONAM! Now if I happened to be lucky enough to have enrolled in the expanded access program at that time, I could have saved my insurance company quite a bit of money for the hospitalization, picc line placement, home nursing… the list goes on and on. Plus my employer would have benefited, not only from my ability to work instead of being sick (considering I was sick for weeks prior to each IV treatment), but maybe it would have kept their insurance premiums from going up astronomically (but that’s just speculation).  Not to mention that since starting the study I have not had a single round of IVs, and my need for oral antibiotics (which are pretty much useless to me anymore) has almost deminished.  I have yet to miss a day of work since starting on this program due to lung issues.

An excellent description of why the FDA has not approved the use of inhaled aztreonam, and has denied appeals since September 2007, is spelled out here by the Cystic Fibrosis Research Institute.  I found it with a simple google search, yes, I do my homework. APPEAL TO FDA FOR APPROVAL OF INHALED AZTREONAM TALKING POINTS FOR ADVOCACY (pdf file)

Perhaps the CFF has been keeping the reasoning quiet as to not step on the FDA’s new board member’s toes? You really don’t want to go pissing off someone saying they aren’t knowledgeable of a topic when they have the authority of denying what you are desperately trying to get approved.  Especially when people’s lives are at stake. -makes sense to me.

Not just anyone can be on the expanded access program (link to details on ClinicalTrials.gov), unfortunately. There are 115 CFF approved care centers and only 65 are participating in the study. Also the study is limited to those who have a lung function with an FEV1 LESS than 50%.  Hence, the Expanded Access Program is not open to those with a lung function above 50%, which rules out most children, adolescents and young adults who definitely could use this drug as a preventative measure, allowing them to keep their highly valued high lung function until a cure comes along.  This program does not account for other people who may not have cf, but still culture pseudomonas, or other bugs that aztreonam could possibly treat.

Yes, I am on this expanded access program. I am getting my inhaled aztreonam for free.  If I didn’t think it could help others, I sure as hell wouldn’t be wishing the FDA would hurry up and approve this drug because then I’d have to open up my wallet.  BUT, out of the goodness in my heart, I wish others could get on this drug, the ones who do not qualify to be in the study, because I know for a fact it has extended my life in years, and improved my quality of life greatly.  I wish the same for my co-cystics, my transplanted buddies, and others who must deal with the nasty vicious Pseudomonas Aeruginosa maybe due to severe lung disease like COPD, PCD, and the list goes on.

I must say again, I just don’t “get it”.

Holy smoke folks!  I never thought I would have this conversation with my docs ever.  Partially because I was reluctant to ask, because I didn’t want to get my hopes up, until I found that special someone to share my life with (yea, I am giving up on that for the time being, until time wears away some more horrible memories).  But my doc said some of the most wonderful things to me.  If I am able to get my lung function just a little bit better, which is very possible and should be easy to do, and keep my diabetes under control, my doc believes I could live a very long and healthy life, which may even include… having a FAMILY!  Oh my! She said the “f” word!  I never really considered this to be an option for me.  So hearing it makes my mind boggle!

I don’t know… maybe doc has been reading my blog and found out that I have been down in the dumps and wanted to lift my spirits about my future… who knows. (by the way, I don’t mind if you read my stuff docs and nurses.. if you are reading this)    But still.  A FAMILY is something that I had never seriously dreamed of.  Much like the people I have dated, I always invisioned myself to have the “expired” stamp on my forehead.  These people have done hell to part of my self-esteem, which you could probably read into in some of my recent posts.  Making me feel like I am less worthy than someone without my genetic mutations.  I look back to see my past wasted with tears caused by people who looked up CF on the web and deemed me lesser than thou.  They hurt me because they were scared of some outdated data.  They were scared I may die (well DUH!).  It is horrible.  And despite this happy news my doctor spoke to me, the tears I have spilled over the years over what others have told me… it is very sad to realize that this cycle will go on, people believing the internet over my word, or even my doctor’s word.  I cry more tears knowing that even though I have been given a blessing of sorts, I may not ever be able to have it just becuase of this damn internet.

What brings us together can also push us apart.

But she still said the “f” word! 

Personally, I am waiting with baited breath for this treatment to hit the market.  Inhaled Aztreonam, AZLI, was expected to be available this past September.  My doc already had plans to put me on it. Due to some changes of FDA board members, this drug is now having to be restudied which means it probably will not hit the market for another two years… a wait too long for some of us.  We are asking for your help in supporting the reconsideration of the application already submitted to the FDA in approving this drug.

I have not yet used AZLI… yet.  But Aztreonam is one of the most effective IV antibiotics I use. Having this drug available in inhaled form could add years to my life.  I have cultured Pseudomonas Aeruginosa for many years (and sometimes many straigns of it).  Having this drug gives me one more weapon against this very harmful bug.

Pseudomonas not only causes horrid infections, it also causes severe irreversible lung damage.  Aztreonam is used as common practice in fighting PA, but right now it is only available via IV.  Having this drug applied directly to the problem, via inhalation, would only make the fight against PA easier and more successful.

Since Aztreonam is only currently available via IV, I only receive it when I am really sick. This inhaled version would be dosed three times a day, 28 days on, 28 days off.  Which basically means I would be fighting my PA with Aztreonam every other month rather than the once to four times per year when I do IVs.  Couple that with the Tobi, inhaled Tobramycin, which I would do on the off months and I would be continuously fighting this evil PA.  I bet that my frequency of hospitalizations would go down given the extra boost I would get from the drug.  Oh my! What life would be like?!!!

Please consider writing the FDA in support of this new drug.
Read additional information about AZLI and the importance of the drug for treating CF.

Cystic Fibrosis

Dear Advocate, Have you or someone you know had a personal experience using Aztreonam Lysine for Inhalation (AZLI)? AZLI is a promising new inhaled antibiotic treatment that appears to help combat the pulmonary symptoms of cystic fibrosis. By sharing your experience using AZLI, you can help the Foundation as it works with the Food and Drug Administration (FDA) to move this new therapy forward. To help, simply: Read our sample letter Write about what AZLI has done for you E-mail your letter to publicpolicy@cff.org by November 28, 2008. Read additional information about AZLI and the importance of the drug for treating CF. Thank you for helping us work with the FDA to advance vital new treatments for cystic fibrosis. Cystic Fibrosis Foundation 6931 Arlington Road Bethesda, MD 20814 (800) FIGHT CF E-mail: publicpolicy@cff.org Web: www.cff.org/GetInvolved/Advocate P.S. Please forward this email to anyone who might be interested in sharing their story about using ALZI.

————————- The CF Foundation is a donor-supported, nonprofit organization committed to controlling and curing cystic fibrosis, a life-threatening genetic disease, and to improving the quality of life for those with CF. To learn more about the Foundation, visit www.cff.org. and subscribe to receive information by e-mail.

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P.S.  Thank you.  ~salty.