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Petition to the FDA calling for clarification Omacetaxine Mepesuccinate, and non-standardized in-vitro diagnostics.

Dear All,

This issue is very vital to all of us.  

In march I went to the FDA and spoke to the ODAC regarding the drug review for Omacetaxine.  I did not speak as a patient on the drug, but on behalf of all CML patients in general to plead the case for the small minority of patients who unfortunately develop the T315I mutation. There were two patients with me who spoke to the advisory committee members to relate their experience as patients diagnosed with the T315I mutation, as well as relate their experience with Omacetaxine.  For many patients Omacetaxine is another lifeline in the journey and battle with CML and could potentially help to remove the “sword of Damocles” hanging over their heads, and indeed our own.


Usually the FDA ODAC committee votes on the clinical efficacy of the drug, but in this case they chose to vote on the lack of a standardized test to determine the presence of the T315I mutation.  This is rather odd as no test currently used to diagnose, or track our treatments are standardized at this time.  That means every test such as:

Bone marrow karyotype
Fluorescent In Situ Hybridization – FISH
Polymerase Chain Reaction testing – PCR

While we commend the work of the FDA and reviewing Omacetaxine, and indeed all of our drugs currently used to treat this disease, we are dismayed by the sudden change of policy.  We are asking the FDA to re-visit their recommendation and allow Omacetaxine to come to market while the company (ChemGenex) works with labs to standardize mutation testing.

This is critical to patients who harbor the T315I.  We do not know what causes this mutation and we cannot really predict who will develop it, so this could be the fate of any one of us.  

Our doctors need more weapons in their armamentarium in the treatment of CML – we are not a one size fits all disease.

We wish to thank Dr. Michael Mauro, Dr. Kantarjian and others for there fantastic work in helping us put together this petition.  

We need to accrue signatures as quickly as possible.

Please read the petition and if you are inclined, please sign it, and pass it along to all your family and friends.  Please also take the time to discuss this with your doctor.

Patients who harbor the T315I mutation are orphans of an orphan disease – please let’s show our solidarity.

This is about standing by clinical efficacy and choices, this is not about brand or drug loyalty.

The CML Society of Canada have joined our resources together with The National CML Society (US) to put this petition forward.  Please review the petition and show your solidarity with those who are afflicted with CML.

Please visit our websites and visit the petition page to read more about this important situation.

This weekend is a holiday weekend in the US, we need your help to keep the momentum going with signatures.  
Cheryl-Anne & Greg Stephens

The Link to the petition: http://www.PetitionOnline.com/FDAOMACE/petition.html

Www.nationalcmlsociety.org    www.cmlsociety.org

Hi Cheryl-anne,

thanks for taking time to post the link to the petition on this forum. I had tried to find the link via your Website but somehow missed it.

I had posted this decision by the FDA on our home page some weeks ago and I must say with some incredulity! I am hoping they will respond favourably and as many people with sign your petition as possible. Decisions by the FDA are followed by EMEA which is the EU regulatory body. In this case I hope they do not follow the FDA to the letter!!!

Sandy ;o)

 

Thank you to all who have signed the petition!

We need more signatures if we intend to make sure the FDA hears our voices.

This issue is of a very critical and vital nature to all of us. Please read on to find out why.

Just two years ago, the FDA gave ChemGenex the green light on Omacetaxine and allowed the drug to be fast tracked, specifically because the drug had showed strong evidence that it was indeed a great benefit to patients harboring the T315I mutation. The FDA had seen the preliminary information and protocols that ChemGenex were using to prove clinical efficacy for this drug. The company, backed by investors then invested millions of dollars in clinical trials and made this drug available to patients who at that time had no other options. 

Yet, on that day in March the FDA suddenly changed their policy, and denied market approval based on the lack of a standardized mutation test. It was a devastating blow to ALL of us!

There is no standardize test for the any of the tests that are currently used to diagnose and monitor treatment for CML, including Karyotyping, FISH or PCR and mutations. Yet for decades, our CML experts have relied on these tests to diagnose and monitor the treatment for CML patients. The mutation test is done in conjunction with the other tests such as FISH and PCR and results from those non standardized tests, corroborates the presence of the T315I mutation that renders current TKI therapy ineffective for this patient population.

This sudden change in policy with the FDA calls not only this drug, but any future drug into question. We feel the FDA need to clarify this sudden change in policy. This petition is about ensuring that our doctors can have a fully stocked armamentarium in choosing the right drug for the right patient in this battle against this insidious disease.

The petition is not about a drug or a company, but it is about clarification on a decision by the FDA that could potentially slow down the availability of new drugs coming to the market.

Please join in solidarity with us and those that we are trying to help.

http://www.petitiononline.com/FDAOMACE/petition.html

For more information please visit The CML Society of Canada website and click on the information about the petition: www.cmlsociety.org And the National CML Society (US) website www.nationalcmlsociety.org

Sign the petition today and make sure your family members and friends do the same. We can do this!

Thank you for your kind consideration,
Cheryl-Anne Simoneau and Greg Stephens