Petition to the FDA calling for clarification on the standing of the drug Omacetaxine Mepesuccinate, and the issue of non-standardized in-vitro diagnostics. sign now

Petition to the FDA calling for clarification on the standing of the drug Omacetaxine Mepesuccinate, and the issue of non-standardized in-vitro diagnostics.

We, CML (Chronic Myelogenous Leukemia) patients, our families, and physicians, formally petition the FDA to clarify its decision regarding the questioning of the specific mutation testing to detect the T315I mutation in the subset of TKI resistant CML patients.

Current diagnostic tests used to monitor CML patients during treatment are:

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

It is our understanding that these tests/assays are not currently standardized, nor are they approved by the FDA. However, they are validated tools in use for decades, well defined and understood in the scientific, medical and patient community. Clinicians who specialize in the treatment of CML rely on the results of these tests, directly and entirely, to guide treatment decisions. Indeed, every single form of drug therapy for CML, currently available commercially and approved by the FDA, was brought to market utilizing information based on the efficacy data relying on the performance of these specific non standardized, non FDA approved tests.

We wish to commend the FDA for their investigation and review of Omacetaxine Mepesuccinate. However, we are concerned about a key factor highlighted in the review, namely the requirement of a standardized test for T315I mutation, and the implications for advancement in the field of therapy for CML.

Omacetaxine Mepesuccinate appears to be a clear potential treatment option for CML patients harboring the T315I mutation, as well as for those with failure of other approved therapies for CML, namely the kinase inhibitors Imatinib, Dasatinib, and Nilotinib. One might consider these patients as orphans within an orphan disease, and treatment options, aside from stem cell transplant, are currently nil.

Detection of abl kinase domain mutations, the disease feature in question and requested to be performed by a standardized assay, is widely available in university and commercial labs. Identification of this specific mutation or a number of other mutations is a significant, defining feature of CML resistant to standard therapy and may be correlated with efficacy of novel therapeutics such as Omacetaxine Mepesuccinate; however, its presence may not be required for activity of novel therapeutics. In the case of Omacetaxine Mepesuccinate, the mechanism of action is independent of Bcr-Abl and thus the T315I mutation is a defining feature of a target population, yet not absolutely required for drug activity. In addition, quantitation of the burden of abnormal or mutation-bearing Bcr-Abl versus wild type Bcr-Abl is not routinely available nor well understood with regards to response to therapy; response to treatments such as Omacetaxine Mepesuccinate rely on the aforementioned standard assays- karyotype, FISH, qPCR for Bcr-Abl in addition to routine blood counts.

Based on the points raised above, clarity is needed in defining standardization requirements for diagnostics in CML. Additionally, with the recent decisions, not in cohesion with current diagnostics used and accepted by the FDA, the unmet need of a limited population, the activity of a novel therapeutic drug, and the relegation of the T315I mutation as a marker defining a target population and NOT a disease response requirement, we would ask that the review of Omacetaxine Mepesuccinate, in particular the requirement for a standardized T315I mutation assay be reconsidered. Most importantly, we ask that the FDA allow Omacetaxine Mepesuccinate to come to market while they collaborate with lab experts on the design of a standardized test.

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Brenda BurnsBy:
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FDA ODAC Committee

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