Positive Results from AML Clinical Research Trial – Midostaurin

Trial Results Pave Way for Breakthrough Therapy Drug Designation Status

Following the Phase III RATIFY clinical trial, Novartis received the Breakthrough Therapy drug designation for the acute myeloid leukemia drug, midostaurin.

The Breakthrough Therapy drug designation allows for the expedition of drug development and review for medicines that treat serious or life-threatening conditions. The drug must have demonstrated quantitative improvement over a current available therapy on at least one clinically determined endpoint. In this case, the compound PKC412 (midostaurin) was compared to the current standard of AML chemotherapy drugs, daunorubicin and cytarabine, with a clinical emphasis on AML cases with a mutated FLT3 gene.

The Phase III RATFIY Clinical Trial

Patients with newly-diagnosed FLT3-mutated Acute Myeloid Leukemia (AML) were enrolled in order to test the investigational compound PKC412, midostaurin. The overall purpose of the clinical research trial was to evaluate the midostaurin or placebo to daunorubicin/cytarabine, the current standard chemotherapy drugs for AML induction.

Patients receiving midostaurin then standard induction and consolidation chemotherapy were compared to those who received only standard induction and consolidation chemotherapy. Overall survival (OS) for patients in the midostaurin treatment group was 74.7 months, while those in the placebo group had an OS of 25.6 months, with no treatment-related deaths observed.

Current AML Treatment

For more than 25 years, acute myeloid leukemia treatment has not seen any progressive changes. 21,000 people in the U.S. are diagnosed with AML each year, with about a third who have an FLT3 gene mutation. This gene mutation has been seen to have worse outcomes and a shorter survival rate in those without the mutation.

Midostaurin is the first drug to show an increase in survival rates with a specific target in the FLT3 gene, a malignancy without any current FDA-approved treatments.

Because midostaurin is expected to be submitted for FDA approval, Novartis has opened a Global Individual Patient Program which will allow approved patients 18 years of age or older with newly-diagnosed FLT3-mutated AML to be considered for midostaurin therapy. Novartis has announced a collaboration with Invivoscribe Technologies, Inc. in order to identify potential patients that meet the requirements of having an FLT3 mutation and with the probability of benefitting from midostaurin treatment.

Hurdles for AML Treatment

About a third of the AML patient population has the hematologic malignancy FLT3 gene mutation. As of yet, an FDA-approved method of targeted treatment has not been found. However, the clinical research trial RATIFY showed to have a significant positive effect on patients with the FLT3 mutated gene, allowing for the FDA to grant midostaurin the Breakthrough Therapy designation. In the future, with a greater sample size research trial, midostaurin could potentially reshape the way we see AML treatment.

Enroll As a Patient For Future Clinical Trials

As stated above, there have not been any significant developments in AML treatment for two and a half decades. The reason we are able to make breakthroughs now is because of patients enrolling in these clinical research trials and helping to improve our knowledge of this condition and how it reacts to trial drugs. Therefore, if you would like to help make a difference in research trials just like this, sign up and enroll as a patient.  We can make a difference. We just need to do it together.