3 replies to this topic

[HPL]

Dear fellow Iclusig patients,

With the latest news about FDA and Iclusig, we have decided to launch the "Iclusig Patient Group". This is a patient advocate group with the primary goal of ensuring that Iclusig will be available to not only us who currently depend on it, but all CML patients. Nobody knows when you may either develop a mutation or build resistance to your existing TKI, and Iclusig needs to be made available to every CML patient as a potential treatment option.

Everyone here knows the risks that have been identified ever since the approval of the drug, but our position is that with an updated label and proper risk mitigation plan, there is a critical need where Iclusig is the best option and must be returned to the market as quickly as possible. Our first action is reaching out to various media outlets as well as the FDA. We need your ideas, input and voices to help ensure that we do our part to ensure that every treatment option is made available to CML patients. This is open to all Iclusig patients globally, since CML knows no borders and the availability is a world wide issue.

I'm reminded lately the Amnesty International slogan "it's better to light a candle, than to curse the darkness". Now is the time for all of us to ensure that the Iclusig patient's voice is heard and considered in these important decisions that need to be made.

More information will follow, but if you're interested in becoming involved, please contact us at IclusigPatientGroup@live.com.

In advance, thanks for being part of the solution!

Beth, Karen and Hans

[PeJ]

I am unfamiliar with Iclusig. Is that a new RX option for CML?

[HPL]

Hi PeJ,

Iclusig is what was originally called AP24534 in trial. Then it was called Ponatinib, and eventually Iclusig It was approved by the FDA back in December 2012, under a fast track approval. The drug was primarily designed for T315I mutation, since that was the an unmet need. It has also shown to be effective in labs against compound mutations, where a cell could develop multiple mutations. It is also approved in Europe, and is undergoing trials in Japan and other locations. The current change is that the FDA has asked Ariad to suspend marketing and sales, since vascular and cardiac events which were already identified as possible side effects, showed up in higher numbers in the during the EPIC trial, as well as subsequent follow up in earlier trials. The EPIC trial was pitting Iclusig against Gleevec as potential first line usage.

It is suspended while the FDA and Ariad figure out a likely more narrow label, along with a risk management strategy, to help better identify which patients may be at risk. After the FDA warnings, Ariad also reduced the recommended dosage from FDA approved 45mg to 15mg if you were responding well, or 30mg if you hadn't gotten a great response yet. Our call to action is around making sure that the drug stays available to CML patients, to meet an otherwise unmet medical need, especially for those with the T315I mutation (which is the most common one), or any other CML patient who may have failed all other available TKI's and is facing a Bone Marrow Transplant. Proper risk management should be followed to weigh risks against efficacy of the drug.

Hope that helps,

Hans

[PeJ]

Thanks, Hans. Very informative.