Ferrirpox Submitted for FDA Approval in the US

I was planning on waiting for the official announcement by ApoPharma, but the Cooley's Anemia Foundation has announced it on their site. I learned this past weekend that Ferriprox (deferiprone, L1) is being submitted for approval to the FDA in the US. After so many delays and while most of the rest of the world has approved L1 and after over 20 years of use in India as Kelfer, we finally see a light at the end of the tunnel.

This site was one of the very first sites online to campaign for the approval of L1 in the US. This dates back to my early involvement at the MSN site and perhaps only Ashish had pre-dated this site in its support for providing the iron chelator L1 in the US. Lisa was on a trial in Philadelphia during the 90's but the bad science that was accepted as fact in the Toronto trials put an end to trials in North America. Lisa flat out told me that she believed the one thing that could save her life was taken away. After all these years, the one chelator that has been proven to be the superior chelator of cardiac iron is finally going to get a chance for FDA approval. This is very timely, as more and more reports are coming in from older patients who have found they cannot use Exjade. Exjade has been promoted as the chelation solution that everyone was waiting for, but I will not be surprised if eventually it loses its prominence in the market as safer and more effective chelators are developed. Frankly, if you have a history of kidney or liver illness, you should be very careful about choosing Exjade and be monitored very closely if you are using it.

Ferrirpox will bring a choice to the patients in the US that is needed, and hopefully we will also see the approval of the Ferriprox oral solution. The oral solution is the ultimate in ease of administration of a chelator and the occurrence of nausea has dropped to 1% with the oral, compared to 16% with the Ferriprox tabs. Only with a variety of chelators can all patients have a chance to survive, and what doctors believe is that you should not plan on spending your entire life on one chelator because they all have long term effects that can be avoided if patients have access to a full choice of chelators. As a long supporter of L1, I am personally very happy about this development and I know my efforts have played a small part in this day's arrival. I wish it had arrived years ago, so many patients could have had a chance to live. Time to move forward.

Andy,

That's definitely a good news,i hope FDA will approve Ferriprox,it has benefited a lot of patients,and as compared to exjade,its much economical.

what doctors believe is that you should not plan on spending your entire life on one chelator because they all have long term effects that can be avoided if patients have access to a full choice of chelators.

By that you mean that we should be switching chelators rather then sticking to only one .

Zaini.

It is still hard to believe. You see, I have been in some trials for L1 15 years ago or even I dont know. I was eight or something like that.

I will definitely switch back on L1 when I have reach the 1000, but then we will three years further.... It's like playing poker (not that I have ever done that).

WOWOWOWOWs its a good news ,,,,,,,

I am glad to know this.  The more options available the better. 

Hi Zaini,  I am assuming that Andy is referring to the fact that chelators can have accumulative affects over time - therefore  it may be best to change chelators over time in order to avoid these affects.  I think that in the past - while people have benefitted from desferal greatly, they have also suffered cumulative side effects from using it for many years.  When there wasn't any other choice one had to choose desferal in order to survive - but now we have the options to switch between chelators to give the body a break from one type of chelator. 

Sharmin

Thanks Sharmin 

But who would like to go back to desferal once they start an oral chelator  .

Zaini.

Hi Zaini,

I totally agree - I wouldn't want to go back to desferal 
But there was some indication in Oakland that the doctors were leaning toward desferal/exjade combination therapy as being ideal if these trials prove to have positive effects.  As we have said in the past - exjade to work at the cellular/tissue level and desferal in the plasma.  He said that exjade should be 7X/week - and desferal 2or3X a week.  This minimizes the dosage of either chelator because you can use a lower dosage of each. 

I think that you have already made some changes with little Z - desferal/L1 and now exjade is giving her a break from the other two. 

Sharmin

I wonder if her doctor would ever let us start Exjade/Desferal therapy,i don't think so,and i am dreading her coming ferritin test  .

Zaini.

Zaini,

I think that once the exjade/desferal studies are complete in the US and safety and effectiveness are established then more doctors will be likely to try it. 

I have heard that the study is going extremely well - most of the patients dropped to ferritin levels below 600 very quickly.  I don't know what Little A will be because his transfusions have just recently started decreasing - so I can only hope that he will drop below 1000 by the end of this year. 

I think that Zaini's ferritin levels will not be sky high because she has been of L1, which works at the cellular level like exjade.  Patients who are on desferal alone or those having organ iron overload should see more increase in ferritin level because they have more iron stored in their organs - so when exjade removes that iron and puts it into the plasma their SF values are high.  Even if they are high, the levels should come down very soon.

Please share the results as soon as you get them, I wish you luck and pray for the best,

Sharmin

Thanks Sharmin 

I hope her ferritin won't be so high,but let's see what happens,she still has 6 tabs left from her first box,so 6 days more before we get all the tests done.

Zaini.

Best of luck my dear!

Sharmin

I heard from my neurologist that the approval/disapproval was going to have a 6 month timeline, give or take. Any news yet?