Follicular Lymphoma Drug Approved by the FDA

Trial Results Show Significant Results Thereby Obtaining FDA Approval

Genentech announced the FDA approval for their anti-CD20 agent drug, Gazyva (obinutuzumab), for follicular lymphoma patients who relapsed after, or are refractory to, a rituximab-containing regimen. This FDA approval was given after significant results were shown during the phase III clinical research trial for their Gazyva drug.

Sandra Horning, MD, Genentech’s CMO and head of Global Product Development stated, “people with follicular lymphoma whose disease returns or worsens despite treatment with a Rituxan-containing regimen need more options because the disease becomes more difficult to treat each time it comes back.”

The FDA also approved of obinutuzumab for use in combination with chlorambucil as a first-line primary treatment for chronic lymphocytic leukemia patients.

The Phase III GADOLIN Clinical Research Trial

It was due to GADOLIN’s study results which allowed for the FDA approval. Obinutuzumab and bendamustine followed by obinutuzumab monotherapy showed to reduce the disease progression by 52% when compared to follicular lymphoma patients who received bendamustine treatment alone.

413 patients with rituximab-refractor indolent non-Hodgkin lymphoma (of which the most common being follicular lymphoma, 321 patients) were studied in the multicenter, open-label GADOLIN trial. Rituximab-refractory classified patients meant that they did not show progression from rituximab monotherapy or rituximab + chemotherapy, or had shown a relapse within 6 months of the last rituximab-based (monotherapy or the combo chemotherapy) dose.

“Gazyva plus bendamustine provides a new treatment option that can be used after relapse to significantly reduce the risk of progression or death,” said Horning.

Details of the GADOLIN Clinical Research Trial

During the clinical research trial, the median patient age was 63 years, about 4 months had passed since their last therapy, and more than 90% of the patients were refractory, had no response, to their last treatment.

Group 1 (experimental arm) received bendamustine and obinutuzumab treatments every 2 months for 2 years. Group 2 (comparator group) received bendamustine monotherapy treatments.

Group 1’s progression-free survival (PFS), the primary endpoint measure, had a median of 29.2 months compared to Group 2’s 13.7 months. At 24.1 months, death risk had reduced by 38% through Group 1’s obinutuzumab regimen compared to Group 2’s monotherapy.

Enroll As a Patient for Future Clinical Trials

Genentech proudly shared their new FDA approval status for follicular lymphoma late February, following their GADOLIN clinical research trial. Such advancements in clinical research are only made possible by the combination of both researchers’ and patients’ efforts. Without the volunteers from the non-Hodgekin lymphoma’s patient population, these critical results would not have been as impactful. Therefore, if you would like to help make a difference in research trials just like this, sign up and enroll as a patient.  Let’s fight the good fight together.

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.